Настройки

Укажите год
-

Небесная энциклопедия

Космические корабли и станции, автоматические КА и методы их проектирования, бортовые комплексы управления, системы и средства жизнеобеспечения, особенности технологии производства ракетно-космических систем

Подробнее
-

Мониторинг СМИ

Мониторинг СМИ и социальных сетей. Сканирование интернета, новостных сайтов, специализированных контентных площадок на базе мессенджеров. Гибкие настройки фильтров и первоначальных источников.

Подробнее

Форма поиска

Поддерживает ввод нескольких поисковых фраз (по одной на строку). При поиске обеспечивает поддержку морфологии русского и английского языка
Ведите корректный номера.
Ведите корректный номера.
Ведите корректный номера.
Ведите корректный номера.
Укажите год
Укажите год

Применить Всего найдено 63187. Отображено 100.
20-11-2009 дата публикации

БИОЧИП ДЛЯ ИССЛЕДОВАНИЯ КЛЕТОК

Номер: RU0000088550U1

1. Биочип для исследования клеток, содержащий подложку с тестовыми участками, в которых могут связываться клетки, отличающийся тем, что в тестовых участках иммобилизованы молекулы веществ, не являющихся антителами, но способных связываться с определенными типами молекул, находящихся на поверхности исследуемых клеток. 2. Биочип по п.1, отличающийся тем, что молекулы веществ, способных связываться с поверхностными молекулами исследуемых клеток, иммобилизованы непосредственно на подложке биочипа. 3. Биочип по п.1, отличающийся тем, что молекулы веществ, способных связываться с поверхностными молекулами исследуемых клеток, иммобилизованы на подложке посредством связей с молекулами одного или нескольких других веществ, находящихся на ее поверхности. 4. Биочип по п.1, отличающийся тем, что на его поверхности иммобилизованы молекулы веществ, ослабляющих связывание исследуемых клеток с материалом подложки. 5. Биочип по п.1, отличающийся тем, что имеет приспособление для крепления в инкубационно-отмывочном устройстве. 6. Биочип по п.1, отличающийся тем, что имеет один или несколько участков, указывающих на порядок считывания результата. 7. Биочип по п.1, отличающийся тем, что имеет разметку, указывающую границы его участков или порядок их расположения. 8. Биочип по п.1, отличающийся тем, что подложка является прозрачной. 9. Биочип по п.1, отличающийся тем, что подложка является светоотражающей. 10. Биочип по п.1, отличающийся тем, что имеет участки контроля получаемого результата. РОССИЙСКАЯ ФЕДЕРАЦИЯ (19) RU (11) 88 550 (13) U1 (51) МПК A61K 48/00 (2006.01) ФЕДЕРАЛЬНАЯ СЛУЖБА ПО ИНТЕЛЛЕКТУАЛЬНОЙ СОБСТВЕННОСТИ, ПАТЕНТАМ И ТОВАРНЫМ ЗНАКАМ (12) ОПИСАНИЕ ПОЛЕЗНОЙ МОДЕЛИ К ПАТЕНТУ (21), (22) Заявка: 2009130448/22, 07.08.2009 (24) Дата начала отсчета срока действия патента: 07.08.2009 (45) Опубликовано: 20.11.2009 (72) Автор(ы): Шишкин Александр Валентинович (RU) (73) Патентообладатель(и): Шишкин Александр Валентинович (RU) R U Адрес для переписки: 426008, г.Ижевск, ул. Карла ...

Подробнее
26-01-2012 дата публикации

Short Interfering Ribonucleic Acid (siRNA) for Oral Administration

Номер: US20120022139A1
Принадлежит: NOVARTIS AG

Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.

Подробнее
02-02-2012 дата публикации

Method of using stem cells to aid in diagnosis

Номер: US20120027678A1
Принадлежит: Individual

The present invention provides a method for the in vitro culture of embryonic stem cells, wherein the stem cells continue to express no antigen or antigen CD117, and mostly remain undifferentiated during culture. The present invention also relates to purified preparations of embryonic stem cells and for uses of embryonic stem cells in treating a wide variety of conditions, diseases and disorders.

Подробнее
02-02-2012 дата публикации

Novel lipids and compositions for the delivery of therapeutics

Номер: US20120027796A1
Принадлежит: Alnylam Pharmaceuticals Inc

The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structures: (Formula (I) or (XXXV)).

Подробнее
02-02-2012 дата публикации

Antibody gene transfer and recombinant aav therefor

Номер: US20120027798A1
Принадлежит: Nationwide Childrens Hospital Inc

The present invention relates generally to the use of recombinant adeno-associated viruses (rAAV) for gene delivery and more specifically to the use of rAAV to deliver antibody genes to target cells in mammals. Administration of rAAV encoding antibodies that neutralize the HIV-1 virus is exemplified.

Подробнее
02-02-2012 дата публикации

Short Interfering Ribonucleic Acid (siRNA) for Oral Administration

Номер: US20120029052A1
Принадлежит: NOVARTIS AG

Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.

Подробнее
09-02-2012 дата публикации

Pharmaceutical composition for treating or preventing cancer by inducing dendritic cell-like differentiation from monocytes to improve anticancer immune activity

Номер: US20120034251A1
Принадлежит: Momotaro Gene Inc

According to the present invention, a composition for inducing or activating dendritic cell-like cells so as to treat or prevent cancer by immunotherapy is provided. Specifically, the following is provided: an agent for activating cancer immunity, which comprises, as an active ingredient, the following REIC protein: (a) a protein consisting of the amino acid sequence shown in SEQ ID NO: 2; or (b) a protein consisting of an amino acid sequence derived from the amino acid sequence shown in SEQ ID NO: 2 by substitution, deletion, or addition of one or more amino acid(s) and having the activity of inducing differentiation from monocytes into dendritic cell-like cells.

Подробнее
09-02-2012 дата публикации

Compositions for the treatment of gluten intolerance and uses thereof

Номер: US20120034299A1
Принадлежит: Glutagen Pty Ltd

The present invention provides compositions for use in the prophylaxis or treatment of a condition arising from gluten intolerance, the compositions including at least partially purified caricain (or a biologically active fragment, analogue or variant thereof) alone or in combination with other suitable enzymes including bromelain, and/or an intestinal extract, as herein described. The present invention also provides methods of using such compositions for the prophylaxis or treatment of a condition arising from gluten intolerance.

Подробнее
09-02-2012 дата публикации

Polyacridine nucleic acid delivery peptide complexes

Номер: US20120035320A1

The present invention provides nucleic acid delivery polyplex complexes and anionic open polyplexes comprising a nucleic acid molecule reversibly bound to one or more of nucleic acid delivery polyplex complexes.

Подробнее
16-02-2012 дата публикации

Ucp4

Номер: US20120041176A1
Принадлежит: Genentech Inc

The present invention is directed to novel polypeptides having homology to certain human uncoupling proteins (“UCPs”) and to nucleic acid molecules encoding those polypeptides. Also provided herein are vectors and host cells comprising those nucleic acid sequences, chimeric polypeptide molecules comprising the polypeptides of the present invention fused to heterologous polypeptide sequences, antibodies which bind to the polypeptides of the present invention, and methods for producing the polypeptides of the present invention.

Подробнее
23-02-2012 дата публикации

TNF-alpha Antagonists Containing IGFBP5

Номер: US20120046217A1

The present invention relates to: TNF-α antagonists containing IGFBP5 protein, variants thereof, or fragments thereof; and the use of the TNF-α antagonists. More specifically, the present invention relates to: a polynucleotide encoding the protein, variants thereof, or fragments thereof; a vector containing the polynucleotide; a transformant containing the vector; and a method for screening a therapeutic agent for TNF-α overexpression-related diseases by checking whether the mutual reaction thereof is facilitated after treating with candidates to the cell expressing the IGFBP5 protein, variants thereof, or fragments thereof, and the TNER1.

Подробнее
23-02-2012 дата публикации

Compositions for targeting conducting airway cells comprising adeno-associated virus constructs

Номер: US20120046349A1
Принадлежит: University of Pennsylvania Penn

An artificial AAV capsid comprising a heterologous conducting airway targeting sequence is provided. The artificial AAV is useful as a targeting moiety, for delivery of heterologous molecules which are associated therewith. The artificial AAV is also useful in the generation of AAV vectors having the artificial capsid. Also described are methods of modifying the native tropism and transduction efficiency of vectors by improving and/or ablating their ability to transduce conducting airway cells. Methods of targeting conducting airway cells and delivering therapeutic and other molecules thereto are also provided.

Подробнее
01-03-2012 дата публикации

Microorganisms for preventing and treating neoplasms accompanying cellular therapy

Номер: US20120052003A9
Автор: Aladar A. Szalay
Принадлежит: Genelux Corp

Provided are methods for using cellular compositions in combination with oncolytic viruses. The methods include administering oncolytic viruses for the inhibition and treatment of tumors caused by administration of cellular therapies, such as stem cell therapies. The methods also include contacting cellular compositions with oncolytic viruses for the removal of neoplastic cells prior to administration of the cellular composition for therapy. Diagnostic methods for monitoring treatment also are provided.

Подробнее
01-03-2012 дата публикации

Cationic poly (amino acids) and uses thereof

Номер: US20120053295A1
Принадлежит: University of Tokyo NUC

The present invention provides an efficient delivery system for a nucleic acid, more specifically, a cationic poly(amino acid) that has a side chain having a plurality of different amine functional groups in a moiety including a cationic group and that has a hydrophobic group introduced into part of the side chain, and a polyion complex (PIC) of the poly(amino acid) and an oligo- or polynucleotide.

Подробнее
08-03-2012 дата публикации

Lipid encapsulated interfering rna

Номер: US20120058188A1
Принадлежит: PROTIVA BIOTHERAPEUTICS INC

The present invention provides compositions and methods for silencing gene expression by delivering nucleic acid-lipid particles comprising a siRNA molecule to a cell.

Подробнее
15-03-2012 дата публикации

Rtef-1 variants and uses thereof

Номер: US20120063994A1

Disclosed are variant RTEF-1 polypeptides having an RTEF-1 amino acid sequence with one or more internal deletions, wherein the polypeptides reduce VEGF promoter activity. Some of the RTEF-1 polypeptides include an amino acid sequence that is at least 80% identical to the contiguous amino acids of 1) amino acids 24 to 47 of SEQ ID NO:15 and 2) each of SEQ ID NOs:16 and 17, but does not comprise the contiguous amino acids of SEQ ID NOs:8, 9, 11, or 12. Also disclosed are nucleic acids encoding the variant RTEF-1 polypeptides of the present invention. Pharmaceutical compositions that include the polypeptides and nucleic acids of the present invention are also disclosed. Methods of inducing cell contact inhibition, regulating organ size, and reducing intracellular YAP activity are also set forth, as well as methods of treating hyperproliferative diseases such as cancer using the pharmaceutical compositions of the present invention.

Подробнее
15-03-2012 дата публикации

Methods of Diagnosing Hypophosphatemic Disorders

Номер: US20120064544A1

The present invention relates to methods of diagnosing hypophosphatemic disorders.

Подробнее
15-03-2012 дата публикации

Rna molecules and therapeutic uses thereof

Номер: US20120065246A1
Автор: Pal Saetrom
Принадлежит: Mina Therapeutics Ltd

The invention relates to double-stranded RNA molecules in which each strand of said molecule possesses: (a) sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof; and (b) sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex; and in which at least one strand of said molecule possesses: (c) a seed region of complementarity to at least one seed site present in a 3′ untranslated region of at least one target mRNA molecule. The invention also relates to an algorithm for the design of a double-stranded RNA molecule in which each strand of said molecule possesses: (a) sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof; and (b) sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex; and in which at least one strand of said molecule possesses: (c) a seed region of complementarity to at least one seed site present in a 3′ untranslated region of at least one target mRNA molecule; wherein said algorithm comprises the steps: (i) input a population of mRNA sequences transcribed from one or more genes of interest; (ii) identify all subsequences of at least 12 nucleotides in length within the population of step (i) which are complementary to another subsequence of at least 12 nucleotides in length in the population; (iii) determine a list of candidate bi-functional double-stranded RNA molecules, said list comprising the double-strand RNA duplexes comprising the two complementary subsequences of step (ii); and (iv) sort the list of candidate double-stranded RNA molecules of step (iii) based on their potential to cause translational suppression.

Подробнее
15-03-2012 дата публикации

Mir-21 promoter driven targeted cancer therapy

Номер: US20120065251A1
Принадлежит: Ramot at Tel Aviv University Ltd

The invention provides a nucleic acid construct comprising a promoter sequence derived from microRNA-21 (miR-21) linked to a nucleic acid sequence encoding an anti-cancer agent, an example of which is a toxin. The constructs of the invention are particularly useful for treating tumors expressing miR-21.

Подробнее
15-03-2012 дата публикации

Genetic modification of targeted regions of the cardiac conduction system

Номер: US20120065253A1
Принадлежит: MEDTRONIC INC

Disclosed are methods and systems for preventing or treating cardiac dysfunction, particularly cardiac pacing dysfunction by genetic modification of the conduction system of the heart. In one embodiment, the invention provides a method of genetically modifying the cells by delivering to the cells one or more coding sequence in a genetic construct capable of modifying the expression of ion channels of the cells.

Подробнее
15-03-2012 дата публикации

Isoform nell-1 peptide

Номер: US20120065574A1
Автор: Chia Soo, Kang Ting
Принадлежит: UNIVERSITY OF CALIFORNIA

This application generally relates to an isoform Nell-1 peptide, compositions thereof, and methods of using the same.

Подробнее
29-03-2012 дата публикации

Treatment of Central Nervous System Disorders

Номер: US20120077741A1
Принадлежит: NeuroNova AB

The invention relates generally to methods of influencing central nervous system cells to produce progeny useful in the treatment of CNS disorders. More specifically, the invention includes methods of exposing a patient suffering from such a disorder to a reagent that modulates the proliferation, migration, differentiation and survival of central nervous system cells. These methods are useful for reducing at least one symptom of the disorder.

Подробнее
29-03-2012 дата публикации

Materials and Methods for the Treatment of Pathological Neovascularization in the Eye

Номер: US20120077870A1

The subject invention provides materials and methods useful in safely and effectively preventing pathological proliferation of blood vessels. The prevention of the over-proliferation of blood vessels according to the subject invention is particularly advantageous for treatment of certain ocular conditions including age-related macular degeneration (AMD), retinopathy of prematurity (ROP) and diabetic retinopathy. In preferred embodiments, the subject invention provides materials and methods for effective treatment of pathological ocular neovascularization using gene therapy. In a specific embodiment the materials and methods of the subject invention can be used to treat AMD.

Подробнее
05-04-2012 дата публикации

Cytotoxic agents

Номер: US20120082716A1
Принадлежит: BTG International Ltd

A method of killing a cell that is lacking in effective p53 protein activity, particularly as compared to wild type, is provided characterised in that it comprises delivering to the cell a single stranded DNA including a portion with at least one base, internally located with respect to any 3′ and 5′ ends of the DNA, that is unbasepaired with another base in a form that is capable of being internalised by the cell.

Подробнее
12-04-2012 дата публикации

Immunotherapeutic agent

Номер: US20120087900A1
Автор: Tai-Gyu Kim

The present invention is directed to adoptive immunotherapy using a lymphocyte in which an antigen-specific receptor and a bioactive material gene such as an IL-2 gene or a water-soluble TGF-beta receptor gene are transferred. The bioactive material is intensively secreted to, for example, a local site of a tumor, thereby reducing systemic side effects as much as possible, and the survival time of the lymphocyte is increased, thereby further improving the effect of the adoptive immunotherapy.

Подробнее
19-04-2012 дата публикации

Process for producing poxviruses and poxvirus compositions

Номер: US20120093780A1
Принадлежит: TRANSGENE SA

The present invention relates to compositions and pharmaceutical compositions comprising poxviruses and more particularly extracellular enveloped viruses. The present invention also relates to a process for producing poxviruses and poxviruses obtained thereof. Moreover, the present invention also relates to the use of said poxvirus and said composition for the preparation of a medicament.

Подробнее
19-04-2012 дата публикации

Implantable liposome embedded matrix composition, uses thereof, and polycaprolactone particles as scaffolds for tissue regeneration

Номер: US20120093914A1
Принадлежит: MOMA THERAPEUTICS

In various embodiments, the present invention describes materials and methods for the local reprogramming of cells in a location where the treatment is applied. The invention can be used to replace lost cells or to restore function to tissue damaged due to disease, injury or genetic defect. In various embodiments, the treatment includes a semisolid hydrogel embedded with liposomes. The liposomes can contain an effector molecule or molecules. When phagocytic cells such as monocytes infiltrate the hydrogel, they encounter the liposomes and incorporate the liposomes carrying the effector molecules into the cells. In some embodiments, the effector molecules can be genetic material encoding the expression of specific proteins such as transcription factors, the expression of which can initiate the reprogramming of the cells. In other embodiments, the effector molecules can induce angiogenesis. In other embodiments, the effector molecules are tumor antigens. The matrix can contain other effector molecules designed to attract specific cells to the matrix. The cells can be released from the matrix as the matrix degrades or by active migration from the matrix. The cells can also remain in the matrix and secret molecules such as proteins and hormones that will diffuse through the matrix material to the surrounding tissue.

Подробнее
19-04-2012 дата публикации

Novel lipids and compositions for the delivery of therapeutics

Номер: US20120095075A1
Принадлежит: Alnylam Pharmaceuticals Inc

The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure:

Подробнее
03-05-2012 дата публикации

Use of LCAT for Treating Anemia and Red Blood Cell Dysfunction

Номер: US20120107298A1
Принадлежит: Alphacore Pharma LLC

Disclosed are methods for treating conditions characterized by anemia or red blood cells dysfunction by administering an agent that increases the level of endogenous LCAT or LCAT activity. Additionally disclosed are methods of treating conditions wherein red blood cells have reduced function in relation to deformability, oxygenation, increased adhesion and aggregability, reduced nitric oxide function, or decreased life-span, increased free cholesterol, or abnormal phospholipid content. Also disclosed are methods for treating conditions characterized by an abnormal concentration of free cholesterol in red blood cells and methods of normalizing the free cholesterol content of red blood cells.

Подробнее
03-05-2012 дата публикации

Amphoteric liposomal compositions for cellular delivery of small rna molecules for use in rna interference

Номер: US20120107389A1
Принадлежит: Individual

The present invention provides method and pharmaceutical composition for efficient delivery of siRNA (small interfering ribonucleic acids) into cultured mammalian cells. In addition, the present invention provides methods and compositions for knocking down the expression of a specific target gene by treating cells with the formulations comprising cationic amphiphile, a neutral colipid and a small RNA molecule. We demonstrate that our method delivers siRNA efficaciously into animal cells for the purpose of RNA interference. The area of medical science that is likely to benefit most from the present invention is RNAi therapeutics.

Подробнее
10-05-2012 дата публикации

Anti-cancer agent, method for inducing apoptosis of cancer cells, and method for screening for anti-cancer agent

Номер: US20120115935A1
Принадлежит: Individual

Disclosed is an anti-cancer agent which can inhibit the progression of cell cycle in the M phase, unlike taxane anti-cancer agents. The anti-cancer agent comprises a compound capable of inhibiting specifically the expression and/or functions of RBM8A depicted in SEQ ID NO: 1, Magoh depicted in SEQ ID NO: 3 or an isoform thereof. The anti-cancer agent can inhibit the expression and/or functions of RBM8A or Magoh in cancer cells to terminate the cell cycle in the M phase, thereby inhibiting the proliferation of the cells. Therefore, the anti-cancer agent can inhibit the proliferation of cancer cells and induce the apoptosis of cancer cells, leading to the cure of cancer.

Подробнее
17-05-2012 дата публикации

Anti-estrogen and immune modulator combinations for treating breast cancer

Номер: US20120121620A1
Автор: David A. Sirbasku
Принадлежит: Individual

Compositions for treating cancers of mucosal tissues including breast, prostate, ovary, colon are disclosed which include various combinations of new or conventional anti-estrogen compounds, aromatase inhibitors, immune modulators, immune inhibitors, immune inhibitor mimicking compounds and steroid or thyroid hormones. Methods of predicting susceptibility of a cancer of mucosal origin to treatment with a composition containing an immune inhibitor or an immune inhibitor mimicking compound are also disclosed. Preferred methods include identifying in a specimen of cancer cells the presence of a Poly-Ig (Fe) receptor or Poly-Ig-like (Fc) receptor capable of binding to an immune inhibitor or an immune inhibitor mimicking compound and of mediating immune inhibition of cancer cell growth.

Подробнее
17-05-2012 дата публикации

Therapeutic regimen for treating cancer

Номер: US20120123186A1
Принадлежит: Genvec Inc

The invention provides a method for treating locally advanced resectable esophageal cancer in a human comprising (a) administering to the human a dose of a pharmaceutical composition comprising (i) a pharmaceutically acceptable carrier and (ii) an adenoviral vector comprising a nucleic acid sequence encoding a human TNF-α and operably linked to a promoter, wherein the dose comprises about 4×10 7 to about 4×10 12 particle units (pu) of adenoviral vector, at least once in a therapeutic period comprising up to about 10 weeks, (b) administering a dose of ionizing radiation to the human over the duration of the therapeutic period, and (c) administering a dose of one or more chemotherapeutics to the human over the duration of the therapeutic period, whereby the locally advanced resectable esophageal cancer in the human is treated.

Подробнее
24-05-2012 дата публикации

Compositions and methods for kinase-mediated cytoprotection and enhanced cellular engraftment and persistence

Номер: US20120128631A1
Автор: Mark A. Sussman

Disclosed are methods of protecting cells, especially non-vascular system, non-hematopoietic cells and tissues, from apoptosis and enhancing their engraftment, survival, and/or persistence by providing enhanced levels of PIM activity for the cell, including PIM-1 activity. Also disclosed are cells that have been engineered to express enhanced levels of PIM kinase, and methods of administering those cells to vertebrates.

Подробнее
24-05-2012 дата публикации

Lipid compositions

Номер: US20120128760A1
Принадлежит: Alnylam Pharmaceuticals Inc

Disclosed herein are lipid compositions comprising a cationic lipid of formula (I), a neutral lipid, a sterol and a PEG or PEG-modified lipid, wherein formula (I) is (F). Also disclosed are methods of producing the cationic lipid of formula (I).

Подробнее
24-05-2012 дата публикации

Transcriptome Transfer Produces Cellular Phenotype Conversion

Номер: US20120129261A1
Принадлежит: University of Pennsylvania Penn

The present invention includes methods for effecting phenotype conversion in a cell by transfecting the cell with phenotype-converting nucleic acid. Expression of the nucleic acids results in a phenotype conversion in the transfected cell. Preferably the phenotype-converting nucleic acid is a transcriptome, and more preferably an mRNA transcriptome.

Подробнее
31-05-2012 дата публикации

Methods for Phototransfecting Nucleic Acids Into Live Cells

Номер: US20120135493A1
Принадлежит: University of Pennsylvania Penn

The present invention includes methods for transferring a multigenic phenotype to a cell by transfecting, preferably by phototransfection, and locally transfecting a cell or a cellular process with a laser while the cell is bathed in a fluid medium comprising two or more nucleic acids, thereby introducing the nucleic acid into the interior of the cell. Expression of the nucleic acids results in a multigenic phenotype in the tranfected cell.

Подробнее
31-05-2012 дата публикации

Treatment of 'c terminus of hsp70-interacting protein' (chip) related diseases by inhibition of natural antisense transcript to chip

Номер: US20120135941A1
Принадлежит: Curna Inc

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of ‘C terminus of HSP70-Interacting Protein’ (CHIP), in particular, by targeting natural antisense polynucleotides of ‘C terminus of HSP70-Interacting Protein’ (CHIP). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of CHIP.

Подробнее
31-05-2012 дата публикации

Carbohydrate conjugates as delivery agents for oligonucleotides

Номер: US20120136042A1
Принадлежит: Alnylam Pharmaceuticals Inc

The present invention provides iRNA agents comprising at least one subunit of the formula (I): wherein: A and B are each independently for each occurrence O, N(R N ) or S; X and Y are each independently for each occurrence H, OH, a hydroxyl protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z′)(Z″)O-nucleoside, —P(Z′)(Z″)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, —P(Z′)(Z″)O-Linker-OP(Z′″)(Z″″)O-oligonucleotide, a nucleotide, an oligonucleotide, —P(Z′)(Z″)-formula (I), —P(Z′)(Z″)— or -Linker-R; R is L G , -Linker-L G , or has the structure shown below: L G is independently for each occurrence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide; R N is independently for each occurrence H, methyl, ethyl, propyl, isopropyl, butyl, or benzyl; and Z′, Z″, Z′″ and Z″″ are each independently for each occurrence O or S.

Подробнее
07-06-2012 дата публикации

Methods and Compositions for Treatment of Muscular Dystrophy

Номер: US20120141441A1
Принадлежит: Leland Stanford Junior University

The present disclosure provides methods for introducing a gene encoding a muscle membrane protein into a cell isolated from a subject to generate a genetically modified cell. The genetically modified cell may be introduced back, e.g., engrafted into the subject. The isolated cell may be additionally modified by introducing into the isolated cell a gene encoding one or more reprogramming transcription factors that induce the cell to form an induced pluripotent stem cell. The genetically modified cell may be differentiated in vitro to form muscle cell precursors before engrafting into the subject. Also provided are compositions comprising autologous cells isolated from a subject which cells comprise a muscle membrane protein gene integrated into a genome attachment site in the genome of the cell. The autologous cell may be an induced pluripotent cell or a mesenchymal stem cell, such as an adipose-derived mesenchymal stem cell (AD-MSC).

Подробнее
07-06-2012 дата публикации

Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap

Номер: US20120142610A1
Принадлежит: OPKO CURNA LLC

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of ‘IQ motif containing GTPase activating protein’ (IQGAP), in particular, by targeting natural antisense polynucleotides of ‘IQ motif containing GTPase activating protein’ (IQGAP). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IQGAP.

Подробнее
21-06-2012 дата публикации

Hybrid Superparamagnetic Iron Oxide Nanoparticles And Polyethylenimine As A Magnetoplex For Gene Transfection

Номер: US20120157626A1
Принадлежит: KAOHSIUNG MEDICAL UNIVERSITY

Disclosed are the nanoparticle and the method for the same, and the preparing method includes steps of mixing polyethylenimine (PEI) with the poly(acrylic acid)-bound iron oxide (PAAIO) to form a PEI-PAAIO polyelectrolyte complex (PEC) and mixing the PEI-PAAIO PEC with genetic material such as plasmid DNA to form the PEI-PAAIO/pDNA magnetic nanoparticle. The PEI-PAAIO/pDNA magnetoplex is highly water dispersible and suitable for long term storage, shows superparamagnetism, low cytotoxicity, high stability and nice transfection efficiency, and thus the PEI-PAAIO PEC can replace PEI as a non-viral gene vector.

Подробнее
05-07-2012 дата публикации

In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages

Номер: US20120172412A1
Принадлежит: Arrowhead Madison Inc

The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.

Подробнее
05-07-2012 дата публикации

Reagents and methods for modulating cone photoreceptor activity

Номер: US20120172419A1

The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.

Подробнее
05-07-2012 дата публикации

Phosphatidyl oligoglycerols

Номер: US20120172631A1
Автор: Hans-Jorg Eibl

In order to form liposomes with a longer half-life in blood, use is made of defined compounds with the general formula (A)

Подробнее
19-07-2012 дата публикации

Novel human p53 splice variant displaying differential transcriptional activity

Номер: US20120183551A1
Принадлежит: Becton Dickinson and Co

Novel human p53 splice variant displaying differential transcriptional activity Described is a nucleic acid molecule encoding a p53 variant characterized in that it is capable of transactivating the p21- and 14-3-3σ-promoter but not the mdm2-, bax- and PIG3-promoter. Preferably, in said p53 variant exon 7, exon 8 and/or exon 9 are partially or entirely deleted. Finally, means for inhibiting the activity of this p53 variant are described which are useful for the therapy of cancer.

Подробнее
26-07-2012 дата публикации

Orally administered bacteria as vehicles for systematic delivery of agents

Номер: US20120189550A1
Принадлежит: University College Cork

The current invention relates to the use of a bacterial species in the preparation of a composition adapted for oral administration for the delivery of an agent to a site in the body. The site in the body may be an organ or a tumour site. The bacterial species is a food grade, non-pathogenic, gram-positive bacteria capable of anaerobic growth.

Подробнее
26-07-2012 дата публикации

Treatment of patients after stent implantation or balloon dilatation and drug eluting stents

Номер: US20120190580A1

The present invention relates to a nucleic acid molecule for use in the treatment or preventive treatment of a patient after stent implantation or balloon dilatation, wherein the nucleic acid molecule is selected from (a) a single-stranded nucleic acid molecule comprising or consisting of the sequence of SEQ ID NO: 1, 2, 3 or 4 or a sequence having at least 90% sequence identity to the sequence of SEQ ID NO: 1, 2, 3 or 4; (b) a hairpin RNA, wherein one of the regions forming the double-stranded portion of said hairpin RNA comprises or consists of the sequence of SEQ ID NO: 1, 2, 3 or 4 or a sequence having at least 90% sequence identity to the sequence of SEQ ID NO: 1, 2, 3 or 4; (c) an at least partially double-stranded RNA comprising two separate single strands, wherein a region within one of the strands, said region being located within the double-stranded portion of said double-stranded RNA, comprises or consists of the sequence of SEQ ID NO: 1, 2, 3 or 4 or a sequence having at least 90% sequence identity to the sequence of SEQ ID NO: 1, 2, 3 or 4; (d) a nucleic acid molecule encoding the nucleic acid molecule of (a) or the RNA (b); and (e) a nucleic acid molecule or a two nucleic acid molecules encoding the two separate single strands of the RNA of (c). The present invention also relates to a drug eluting stent comprising the nucleic acid molecule according to the invention.

Подробнее
26-07-2012 дата публикации

Methods and compositions to reduce oxidative stress

Номер: US20120190731A1
Автор: Louis M. Messina
Принадлежит: University of Massachusetts UMass

The invention relates to therapeutic applications for compositions that reduce the level of oxidative stress on cells in vivo or in vitro. The invention describes methods for improving the therapeutic properties of stem cells. The invention also provides combination therapies that are useful to balance the oxidative microenvironment of cells in vivo or in vitro.

Подробнее
26-07-2012 дата публикации

Methods and compositions for gene correction

Номер: US20120192301A1
Принадлежит: Sangamo Therapeutics Inc

Disclosed herein are methods and compositions for correction and/or mutation of genes associated with Parkinson's Disease as well as clones and animals derived therefrom.

Подробнее
02-08-2012 дата публикации

Modulation of Synaptic Maintenance

Номер: US20120195880A1
Принадлежит: Leland Stanford Junior University

C1q is shown to be expressed in neurons, where it acts as a signal for synapse elimination. Methods are provided for protecting or treating an individual suffering from adverse effects of synapse loss. These findings have broad implications for a variety of clinical conditions, including Alzheimer's disease.

Подробнее
02-08-2012 дата публикации

Phosphodiesterase 4 inhibitors for cognitive and motor rehabilitation

Номер: US20120196898A1
Принадлежит: Individual

The present invention provides methods of improving cognitive and motor deficits associated with central nervous system (CNS) disorder or condition in an animal. The methods comprise a general administration of phosphodiesterase 4 inhibitors and optionally training the animal under conditions sufficient to produce an improvement in performance.

Подробнее
16-08-2012 дата публикации

Adipose-derived stem cells and lattices

Номер: US20120208274A1
Принадлежит: Individual

The present invention provides adipose-derived stem cells (ADSCs), adipose-derived stem cell-enriched fractions (ADSC-EF) and adipose-derived lattices, alone and combined with the ADSCs of the invention. In one aspect, the present invention provides an ADSC substantially free of adipocytes and red blood cells and clonal populations of connective tissue stem cells. The ADSCs can be employed, alone or within biologically-compatible compositions, to generate differentiated tissues and structures, both in vivo and in vitro. Additionally, the ADSCs can be expanded and cultured to produce molecules such as hormones, and to provide conditioned culture media for supporting the growth and expansion of other cell populations. In another aspect, the present invention provides a adipose-derived lattice substantially devoid of cells, which includes extracellular matrix material from adipose tissue. The lattice can be used as a substrate to facilitate the growth and differentiation of cells, whether in vivo or in vitro, into anlagen or even mature tissues or structures.

Подробнее
16-08-2012 дата публикации

Optimized Placement of Cannula for Delivery of Therapeutics to the Brain

Номер: US20120209110A1
Принадлежит: UNIVERSITY OF CALIFORNIA

Methods and systems are provided for improved delivery of agents to targeted regions of the brain, by the use of placement coordinates that provide for optimal placement of delivery cannula. By optimizing the cannula placement, reproducible distribution of infusate in the targeted region of the brain is achieved, allowing a more effective delivery of therapeutics to the brain.

Подробнее
13-09-2012 дата публикации

Anti-C5A Binding Moieties with High Blocking Activity

Номер: US20120231008A1

The present invention relates to binding moieties that specifically bind to a conformational epitope of C5a, in particular human C5a. Preferred binding moieties are anti-C5a antibodies that bind to this conformational epitope. The binding moieties described herein are useful as active agents in pharmaceutical compositions for the treatment and prevention of various acute and chronic diseases, in particular acute inflammatory diseases, such as the systemic inflammatory response syndrome (SIRS), and different degrees of sepsis including sepsis, severe sepsis, and septic shock.

Подробнее
13-09-2012 дата публикации

Regulation of neurotransmitter release through anion channels

Номер: US20120232127A1

A novel use of anion channels, preferably Ca 2+ -activated anion channels (CAACs), in regulating release of neurotransmitters from neurons and/or astrocytes is provided. More specifically, CAAC activity regulators, agents for regulating neurotransmitter release comprising such CAAC activity regulators, and methods of screening agents for regulating neurotransmitter release using CAAC as a target.

Подробнее
27-09-2012 дата публикации

Tusc2 therapies

Номер: US20120244209A1
Принадлежит: University of Texas System

A method for predicting a subject's response to a TUSC2 therapy is provided. In particular, a subject's response is predicted based on the proportion of cancers cells that are apoptotic. Also provided is a method of treating a subject previously predicted to have a favorable response with a TUSC2 therapy. Methods for treating cancer by administration of a TUSC2 therapeutic in conjunction with an EGFR inhibitor and/or a protein kinase inhibitor are also disclosed. Kits and reagents for use in TUSC2 therapy are provided.

Подробнее
04-10-2012 дата публикации

Method and Composition to Increase Radiation-Induced Tumor Therapeutic Effects

Номер: US20120252883A1

Disclosed herein are methods and compositions for treating cancer by increasing radiation-induced damage to cancer without increasing radiation-induced side effects by increasing secretory ASMase levels specifically in tumor endothelium, and inducing apoptosis of tumor endothelial cells by treating the tumor with radiation. ASMase levels are increased in tumor endothelium by administration of a recombinant DNA construct comprising a region coding for a functional ASMase linked to particular transcriptional regulatory sequences that confer tissue-specific expression of ASMase.

Подробнее
04-10-2012 дата публикации

Compositions and Methods for Identifying Agents Which Modulate PTEN Function and PI-3 Kinase Pathways

Номер: US20120253020A1
Автор: Donald L. Durden
Принадлежит: Individual

Methods are provided for the identification, biochemical characterization and therapeutic use of agents which impact PTEN, p53, PI-kinase and AKT mediated cellular signaling.

Подробнее
04-10-2012 дата публикации

Inducible small rna expression constructs for targeted gene silencing

Номер: US20120255045A1

The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic, particularly mam

Подробнее
11-10-2012 дата публикации

Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids

Номер: US20120258046A1
Автор: Thorsten Mutzke
Принадлежит: Individual

The present invention is directed to (the use of) a solution containing at least one nucleic acid (sequence) and free mannose for lyophilization, transfection and/or injection, particularly of RNA and mRNA. The inventive solution exhibits a positive effect on stabilization of the nucleic acid (sequence) during lyophilization and storage but also leads to a considerable increase of the transfection efficiency of a nucleic acid. It thus also increases in vivo expression of a protein encoded by such a nucleic acid upon increased transfection rate. The present invention is furthermore directed to a method of lyophilization using the mannose-containing solution, to pharmaceutical compositions, vaccines, kits, first and second medical uses applying such a mannose-containing solution and/or a nucleic acid (sequence) lyophilized or resuspended with such a solution.

Подробнее
11-10-2012 дата публикации

Novel Therapeutical Tools and Methods for Treating Blindness

Номер: US20120258530A1

The present inventions relates to the use of an isolated nucleic acid molecule comprising a nucleotide sequence coding for a hyperpolarizing light-gated ion channel or pump gene from an archeon or for a light-active fragment of said gene, or the nucleotide sequence complementary to said nucleotide sequence, for treating or ameliorating blindness. The light-gated ion channel or pump gene can be a halorhodopsin gene.

Подробнее
01-11-2012 дата публикации

Oncolytic vaccinia virus cancer therapy

Номер: US20120276053A1
Автор: David Kirn
Принадлежит: Individual

Embodiments of the invention are directed methods that include a thymidine kinase deficient vaccinia virus. The methods include administering the vaccinia virus at increased viral concentrations. Further aspects of the invention include methods for inducing oncolysis or collapse of tumor vasculature in a subject having a tumor comprising administering to a subject at least 1×10 8 infectious viral particles of a TK-deficient, GM-CSF-expressing, replication-competent vaccinia virus vector sufficient to induce oncolysis of cells in the tumor.

Подробнее
01-11-2012 дата публикации

Nucleic acid sequences encoding and compositions comprising ige signal peptide and/or il-15 and methods for using the same

Номер: US20120276142A1
Принадлежит: Individual

Fusion proteins and nucleic acid molecules encoding fusion proteins are disclosed. Fusion proteins comprising non-IL-15 signal peptide linked to IL-15 protein sequences and fusion proteins comprising an IgE signal peptide linked to non-IgE protein sequences are disclosed. Vectors comprising such nucleic acid molecules; and to host cells comprising such vectors are disclosed as well as recombinant vaccines and live attenuated pathogens encoding fusion proteins, and methods of using the same, are disclosed. The immunomodulatory effect following delivery of IL-15 and CD40L, with or without immunogens, is disclosed as are various nucleic acid molecules and compositions thereof used for delivering such proteins and methods of using such compositions.

Подробнее
08-11-2012 дата публикации

Production of closed linear dna

Номер: US20120282283A1
Автор: Vanessa Hill
Принадлежит: Individual

An in vitro process for the production of closed linear deoxyribonucleic acid (DNA) comprises (a) contacting a DNA template comprising at least one protelomerase target sequence with at least one DNA polymerase in the presence of one or more primers under conditions promoting amplification of said template; and (b) contacting amplified DNA produced in (a) with at least one protelomerase under conditions promoting production of closed linear DNA. A kit provides components necessary in the process.

Подробнее
08-11-2012 дата публикации

Silica particle including a molecule of interest, method for preparing same and uses thereof

Номер: US20120283379A1

What is provided includes a nanoparticle of porous silica, incorporating at least one molecule of interest, the silica network inside said nanoparticle being functionalized by at least one group capable of setting up an ionic and/or hydrogen non-covalent bond with the molecule of interest, whereby the molecule(s) of interest is(are) linked to the silica network solely by non-covalent bonds. In addition, a method for preparing said silica particle and uses thereof is provided.

Подробнее
15-11-2012 дата публикации

Regulation of t cell-mediated immunity by tryptophan

Номер: US20120288472A1
Автор: Andrew Mellor, David Munn
Принадлежит: GEORGIA HEALTH SCIENCES UNIVERSITY

A mechanism of macrophage-induced T cell suppression is the selective elimination of tryptophan and/or increase in one or more tryptophan metabolites within the local macrophage microenvironment Expression of IDO can serve as a marker of suppression of T cell activation, and may play a significant role in allogeneic pregnancy and other types of transplantation. Inhibitors of IDO can be used to activate T cells. Inhibiting tryptophan degradation, or supplementing tryptophan concentration, can be used in addition to, or in place of, inhibitors of IDO. Increasing tryptophan degradation (thereby, decreasing tryptophan concentration and increasing tryptophan metabolite concentration), for example, by increasing IDO concentration or IDO activity, can suppress T cells. One can manipulate local tryptophan concentrations, and/or modulate the activity of the high affinity tryptophan transporter, and/or administer tryptophan degrading enzymes. Regulation can be further manipulated using cytokines such as MCSF, IFNγ, alone or in combination with antigen or other cytokines.

Подробнее
15-11-2012 дата публикации

SDF-1 Delivery For Treating Ischemic Tissue

Номер: US20120289585A1

A method of treating a cardiomyopathy in a subject includes administering directly to or expressing locally in a weakened, ischemic, and/or peri-infarct region of myocardial tissue of the subject an amount of SDF-1 effective to cause functional improvement in at least one of the following parameters: left ventricular volume, left ventricular area, left ventricular dimension, cardiac function, 6-minute walk test, or New York Heart Association (NYHA) functional classification.

Подробнее
22-11-2012 дата публикации

Reporter vector presenting extracellular binding capacity to metallic compounds

Номер: US20120295274A1
Принадлежит: Individual

According to one embodiment, a reporter vector presenting an extracellular binding capacity to metallic compounds contains a nucleotide sequence exhibiting a promoter activity depending on a specific condition, a nucleotide sequence encoding a metallic compound-binding peptide presented extracellularly, and a nucleotide sequence encoding transcription termination signals.

Подробнее
22-11-2012 дата публикации

Gene Therapy Using Transposon-Based Vectors

Номер: US20120297493A1

Methods and compositions are presented for the administration of transposon-based vectors to an animal or human to provide gene therapy to the animal or human.

Подробнее
29-11-2012 дата публикации

Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof

Номер: US20120304321A1
Принадлежит: CELLECTIS SA

A monomer of an I-CreI meganuclease variant wherein said monomer when in dimeric form binds and cleaves DNA.

Подробнее
06-12-2012 дата публикации

Antibodies specific for claudin 6 (cldn6)

Номер: US20120308478A1

The present invention provides antibodies useful as therapeutics for treating and/or preventing diseases associated with cells expressing Claudin-6 (CLDN6), including tumor-related diseases such as ovarian cancer, lung cancer, gastric cancer, breast cancer, hepatic cancer, pancreatic cancer, skin cancer, malignant melanoma, head and neck cancer, sarcoma, bile duct cancer, cancer of the urinary bladder, kidney cancer, colon cancer, placental choriocarcinoma, cervical cancer, testicular cancer, and uterine cancer.

Подробнее
06-12-2012 дата публикации

Methods and Compositions for Gene Inactivation

Номер: US20120308528A1
Принадлежит: Sangamo Biosciences Inc

Disclosed herein are methods and compositions for inactivating CCR-5 genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs, such as adenovirus (Ad) vectors, and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided.

Подробнее
06-12-2012 дата публикации

Compositions and methods for topical delivery of oligonucleotides

Номер: US20120309815A1
Принадлежит: ISIS PHARMACEUTICALS INC

The present invention relates to compositions and methods which enhance the delivery of nucleic acids and other nucleosidic moieties via topical routes of administration. The invention relates to the use of an aqueous solution to preferentially deliver nucleic acids preferentially to hair follicles. The invention relates to a method of inhibiting hair growth comprising administration of a nucleic acid preferentially to a hair follicle.

Подробнее
13-12-2012 дата публикации

Tumor suppressor designated ts10q23.3

Номер: US20120315631A1

A specific region of chromosome 10 (10q23.3) has been implicated by series of studies to contain a tumor suppressor gene involved in gliomas, as well as a number of other human cancers. One gene within this region was identified, and the corresponding coding region of the gene represents a novel 47 kD protein. A domain of this product has an exact match to the conserved catalytic domain of protein tyrosine phosphatases, indicating a possible functional role in phosphorylation events. Sequence analyses demonstrated the a number of exons of the gene were deleted in tumor cell lines used to define the 10q23.3 region, leading to the classification of this gene as a tumor suppressor. Further analyses have demonstrated the presence of a number of mutations in the gene in both glioma and prostate carcinoma cells. Methods for diagnosing and treating cancers related to this tumor suppressor, designated as TS10q23.3, also are disclosed.

Подробнее
13-12-2012 дата публикации

Chiral Diacylhydrazine Ligands for Modulating the Expression of Exogenous Genes via an Ecdysone Receptor Complex

Номер: US20120316066A1
Принадлежит: Intrexon Corp

The present invention provides diacylhydrazine ligands and chiral diacylhydrazine ligands for use with ecdysone receptor-based inducible gene expression systems. Thus, the present invention is useful for applications such as gene therapy, large scale production of proteins and antibodies, cell-based screening assays, functional genomics, proteomics, metabolomics, and regulation of traits in transgenic organisms, where control of gene expression levels is desirable. An advantage of the present invention is that it provides a means to regulate gene expression and to tailor expression levels to suit the user's requirements.

Подробнее
13-12-2012 дата публикации

Novel nucleic acid prodrugs and methods of use thereof

Номер: US20120316224A1
Принадлежит: Ontorii Inc

Described herein are nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties. Also described herein are methods of making and using nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties.

Подробнее
20-12-2012 дата публикации

Anti-c4.4a antibodies and uses thereof

Номер: US20120321619A1
Принадлежит: Bayer Intellectual Property GmbH

The present invention provides recombinant antigen-binding regions and antibodies and functional fragments containing such antigen-binding regions that are specific for the membrane-anchored, 29 kDa C4.4a polypeptide, which is over expressed in several tumors, e.g. lung, colorectal, pancreas, prostate, renal and breast cancer. These antibodies, accordingly, can be used to treat these and other disorders and conditions. Antibodies of the invention also can be used in the diagnostics field, as well as for further investigating the role of C4.4a in the progression of disorders associated with cancer. The invention also provides nucleic acid sequences encoding the foregoing antibodies, vectors containing the same, pharmaceutical compositions and kits with instructions for use.

Подробнее
20-12-2012 дата публикации

Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene

Номер: US20120322853A1
Принадлежит: Curna Inc

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Pancreatic Developmental gene, in particular, by targeting natural antisense polynucleotides of a Pancreatic Developmental gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Pancreatic Developmental genes.

Подробнее
27-12-2012 дата публикации

Therapeutic agent for fibroid lung

Номер: US20120328694A1
Принадлежит: Nitto Denko Corp

Disclosed are: a substance transfer carrier to an extracellular matrix-producing cell in the lung, which comprises a retinoid; a therapeutic agent for fibroid lung, which utilized the carrier; and a preparation kit of the therapeutic agent.

Подробнее
17-01-2013 дата публикации

Lineage-Restricted Neuronal Precursors

Номер: US20130017179A1

A cell population has been identified and isolated that can differentiate into multiple neuronal phenotypes, but cannot differentiate into glial phenotypes. This mammalian CNS neuron-restricted cell expresses highly polysialated or embryonic neural cell adhesion molecule (E-NCAM) and is morphologically distinct from neuroepithelial stem cells (NEP cells) and spinal glial progenitors derived from embryonic day 10.5 spinal cord. Methods for isolating these cells and uses thereof are also disclosed.

Подробнее
17-01-2013 дата публикации

Novel modified galectin 9 proteins and use thereof

Номер: US20130017998A1
Принадлежит: Individual

A recombinant galectin 9 (rGal 9) is provided which exhibits an immune system-mediated action and a direct action on tumor cells (i.e., activity of inducing the intercellular adhesion and apoptosis of the tumor cells), thereby potent in inducing the inhibition of cancer metastasis and reduction. Moreover, the rGal 9 exerts no efficacy on non-activated lymphocytes but can induce apoptosis in activated T cells, in particular, CD4-positive T cells causing an excessive immune response.

Подробнее
31-01-2013 дата публикации

Immune Effector Cells Pre-Infected with Oncolytic Virus

Номер: US20130028875A1
Принадлежит: Leland Stanford Junior University

Compositions and methods are provided for the treatment of cancer. An immune effector cell population is pre-infected with an oncolytic virus. The combined therapeutic is safe and highly effective, producing an enhanced anti-tumor effect compared to either therapy alone. The methods of the invention thus provide for a synergistic effect based on the combined biotherapeutics.

Подробнее
31-01-2013 дата публикации

Diagnosis and treatment of multiple sulfatase deficiency and other sulfatase deficiencies

Номер: US20130028881A1
Принадлежит: Shire Human Genetics Therapies Inc

This invention relates to methods and compositions for the diagnosis and treatment of Multiple Sulfatase Deficiency (MSD) as well as other sulfatase deficiencies. More specifically, the invention relates to isolated molecules that modulate post-translational modifications on sulfatases. Such modifications are essential for proper sulfatase function.

Подробнее
31-01-2013 дата публикации

Cerberus/coco derivatives and uses thereof

Номер: US20130029908A1
Автор: Jasbir Seehra, John Knopf
Принадлежит: Acceleron Pharma Inc

The invention relates to Cerberus/Dan/Gremlin polypeptides or variants thereof for use in treating a variety of disorders associated with myostatin, nodal and GDF-11. Preferred polypeptides are Coco or Cerberus derivatives.

Подробнее
07-02-2013 дата публикации

Lipid formulated dsrna targeting the pcsk9 gene

Номер: US20130035371A1
Принадлежит: Individual

This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene.

Подробнее
14-02-2013 дата публикации

Immunogenic pote peptides and methods of use

Номер: US20130039936A1

POTE has recently been identified as a tumor antigen expressed in a variety of human cancers, including colon, ovarian, breast, prostate, lung and pancreatic cancer. Described herein are immunogenic POTE polypeptides, including modified POTE polypeptides, that bind MHC class I molecules. The immunogenic POTE polypeptides are capable of inducing an immune response against POTE-expressing tumor cells. Thus, provided herein is a method of eliciting an immune response in a subject, such as a subject having a type of cancer that expresses POTE.

Подробнее
07-03-2013 дата публикации

Adeno-associated virus serotype i nucleic acid sequences, vectors and host cells containing same

Номер: US20130059289A1
Принадлежит: University of Pennsylvania Penn

The nucleic acid sequences of adeno-associated virus (AAV) serotype 1 are provided, as are vectors and host cells containing these sequences and functional fragments thereof. Also provided are methods of delivering genes via AAV-1 derived vectors.

Подробнее
14-03-2013 дата публикации

Novel cationic lipids and methods of use thereof

Номер: US20130064894A1
Принадлежит: PROTIVA BIOTHERAPEUTICS INC

The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.

Подробнее
14-03-2013 дата публикации

Nlk as a marker for diagnosis of liver cancer and as a therapeutic agent thereof

Номер: US20130065945A1
Автор: Suk Woo Nam

A novel marker for diagnosis of liver cancer and use thereof are provided. To be specific, a marker for diagnosis of liver cancer using over-expression of NLK (neuro-like kinase) in liver cancer cell is provided, along with a composition for diagnosis of liver cancer, a kit, a microarray, and a method for diagnosing liver cancer using the marker. Additionally, a method for screening a substance to prevent or treat liver cancer by decreasing expression of the marker gene or protein, and a composition for preventing or treating liver cancer including such substance are provided. Accordingly, the NLK gene can be efficiently used as a target for diagnosis and treatment of liver cancer.

Подробнее
14-03-2013 дата публикации

Tumor-specific promoter and oncolytic virus vector comprising the same

Номер: US20130065952A1

Provided are tumor-specific promoters, oncolytic virus vectors and a pharmaceutical composition comprising the virus vector. The virus vector comprising the novel tumor-specific promoter shows excellent oncolytic effects on tumor cells, and thus it is useful for treating a cancer.

Подробнее
14-03-2013 дата публикации

Ucp4

Номер: US20130066052A1
Принадлежит: Genentech Inc

The present invention is directed to novel polypeptide, designated in the present application as “UCP4” (SEQ ID NO: 1), having homology to certain human uncoupling proteins (“UCPs”) and to nucleic acid molecules encoding those polypeptides. Also provided herein are vectors and host cells comprising those nucleic acid sequences, chimeric polypeptide molecules comprising the polypeptides of the present invention fused to heterologous polypeptide sequences, antibodies which bind to the polypeptides of the present invention, and methods for producing the polypeptides of the present invention.

Подробнее
21-03-2013 дата публикации

INSULIN-LIKE GROWTH FACTOR II (IGF-II) BINDING FACTORS

Номер: US20130071366A1
Принадлежит: Cancer Research Technology Limited

This invention relates to modified IGF-II binding domains of the Insulin-like Growth Factor 2 Receptor (IGF2R) which have enhanced binding affinity for IGF-II relative to the wild type IGF-II binding domain. Suitable IGF-II binding domains may be modified, for example, by substituting residue E1544 for a non-acidic residue. These modified domains may be useful in the sequestration of Insulin-like Growth Factor II (IGF-II), for example, in the treatment of cancer. 125-. (canceled)26. A mutant IGF-II binding domain comprising an amino acid sequence that has at least 80% sequence identity with residues 1511 to 1650 of human IGF2R ,wherein residue E1544 is substituted for a non-acidic residue, andwherein said binding domain binds IGF-II with increased affinity relative to residues 1511 to 1650 of human IGF2R.27. The mutant IGF-II binding domain of claim 26 , wherein the binding domain does not bind or does not substantially bind to IGF1.28. The mutant IGF-II binding domain of claim 26 , wherein the mutated residues are mutated by substitution claim 26 , insertion or deletion.29. The mutant IGF-II binding domain of claim 26 , wherein residue E1544 is substituted for a basic residue.30. The mutant IGF-II binding domain of claim 29 , wherein residue E1544 is substituted for K.31. The mutant IGF-II binding domain of claim 29 , wherein residue E1544 is substituted for R.32. The mutant IGF-II binding domain of claim 29 , wherein residue E1544 is substituted for H.33. The mutant IGF-II binding domain of claim 29 , wherein residue E1544 is substituted for S.34. The mutant IGF-II binding domain of claim 26 , wherein residues F1567 and I1572 are not mutated.35. The mutant IGF-II binding domain of claim 34 , wherein residue T1570 is not mutated.36. The mutant IGF-II binding domain of claim 34 , wherein residues P1597 and P1599 are not mutated.37. The mutant IGF-II binding domain of claim 26 , wherein the binding domain consists of the amino acid sequence of residues 1511 to 1650 ...

Подробнее
21-03-2013 дата публикации

Amphiphilic Cationic Polymers and Methods of Use Thereof

Номер: US20130071444A1

Amphiphilic cationic polymers comprising a biocompatible amphiphile linked to an organic cation are provided. The polymers complex with therapeutic agents and facilitate delivery of such therapeutic agents, particularly therapeutic nucleic acids, both in vitro and in vivo. Accordingly, the invention further provides methods of facilitating delivery of therapeutic and/or diagnostic agents to a cell and methods of treating a condition, such as a disease or infection, in an organism using the amphiphilic cationic polymers of the invention. 1. A composition comprising a therapeutic agent in combination with an amphiphilic cationic polymer , wherein the amphiphilic cationic polymer comprises a biocompatible amphiphile linked to an organic cation , and wherein the biocompatible amphiphile and the organic cation are linked by a biodegradable linker.3. The composition of claim 1 , wherein the biocompatible amphiphile is an amphiphilic block copolymer.5. The composition of claim 1 , wherein the biocompatible amphiphile has a hydrophilic-lipophilic balance (HLB) of about 10 to about 26.6. The composition of claim 1 , wherein the biocompatible amphiphile has a size of about 1000 Da to about 10000 Da.7. The composition of claim 1 , wherein the organic cation is an amine.8. The composition of claim 7 , wherein the amine is selected from the group consisting of polyethylenimine (MW≦2000 Da) claim 7 , dendrimer (MW≦3000 Da) claim 7 , bis-aminopropyl piperazine (BAPP) claim 7 , and arginine.9. The composition of claim 1 , wherein the biodegradable linker is selected from the group consisting of an esteramine and a carbamate.10. The composition of claim 1 , wherein the therapeutic agent is a nucleic acid.11. The composition of claim 10 , wherein the nucleic acid is an oligonucleotide.12. The composition of claim 1 , wherein the composition forms a homogenous collection of particles having a diameter of about 50 nm to about 300 nm.13. A pharmaceutical composition comprising a ...

Подробнее
21-03-2013 дата публикации

Saposin-a derived peptides and uses thereof

Номер: US20130072425A1
Автор: Randolph Watnick
Принадлежит: Childrens Medical Center Corp

The invention relates to isolated peptides and chimeric polypeptides derived from Saposin A that have anti-angiogenic activity. These peptides are small, consisting essentially of at least 10 consecutive amino acid residues from the 31st-50 th amino acid residue of Saposin A. The invention also relates to the use of these isolated peptides and chimeric polypeptides in compositions for the treatment, prevention, and inhibition of angiogenesis-related diseases and disorders such as cancer and cancer metastasis.

Подробнее
21-03-2013 дата публикации

Treatment of methionine sulfoxide reductase a (msra) related diseases by inhibition of natural antisense transcript to msra

Номер: US20130072546A1
Принадлежит: Curna Inc

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Methionine Sulfoxide Reductase A (MSRA), in particular, by targeting natural antisense polynucleotides of Methionine Sulfoxide Reductase A (MSRA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of MSRA.

Подробнее
21-03-2013 дата публикации

Scalable Manufacturing Platform for Viral Vector Purification and Viral Vectors So Purified for Use in Gene Therapy

Номер: US20130072548A1
Принадлежит:

Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use. 1. A method for purifying bona fide AAV vector particles comprising a transgene encoding a therapeutic protein or fragment thereof from an AAV preparation comprising AAV vector particles , empty capsids and host cell impurities , thereby providing an AAV product substantially free of AAV empty capsids , said method comprising:a) harvesting cells comprising recombinant AAV;b) concentrating said cells via Tangential Flow Filtrationc) lysing said cells by microfluidization to form a lysate;d) filtering, thereby clarifying the lysate of step c);e) purifying AAV particles by Ion Exchange Column Chromatography and optionally concentrating column eluate by Tangential Flow Filtration;f) mixing said eluate with cesium chloride and subjecting said mixture to centrifugation, thereby forming a gradient;g) collecting viral particles separated in step f) and subjecting the same buffer exchange by Tangential Flow Filtration;h) formulating purified AAV particles with surfactant to provide an AAV particle formulation;i) filtering said formulation to remove any remaining impurities, wherein said bona fide AAV vector particles are present in said AAV product in an amount of at least 95%.2. The method of claim 1 , wherein said AAV vector particles are present at a concentration of 100 mg/mL.3. The method of claim 1 , wherein said AAV particles of step i) are present at a concentration of 10particles per mL.4. The method of claim 1 , wherein said AAV particles of step i) are present at a concentration of 10particles per mL.5. The method of claim 1 , wherein said AAV particles of step i) are present at a concentration of 10particles per mL.6. The method of claim 1 , wherein said AAV vector particles are derived from an AAV selected from the group consisting of AAV1 claim 1 , AAV2 claim 1 , AAV5 claim 1 , AAV6 claim 1 , AAV8 and AAV9. ...

Подробнее
21-03-2013 дата публикации

Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure

Номер: US20130072671A1
Принадлежит: Leids Universitair Medisch Centrum LUMC

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.

Подробнее
28-03-2013 дата публикации

Live, oral vaccine for protection against shigella dysenteriae serotype 1

Номер: US20130078274A1
Принадлежит: US Government

The invention relates to Salmonella typhi Ty21a comprising core-linked Shigella dysenteriae serotype 1 O-specific polysaccharide (O-Ps) and DNA encoding O antigen biosynthesis, said DNA selected from the group consisting of: a) the DNA sequence set out in any one of SEQ ID NOs: 1 and 2 and species homologs thereof; b) DNA encoding Shigella dysenteriae serotype 1 polypeptides encoded by any one of SEQ ID NOs: 1 and 2, and species homologs thereof; and c) DNA encoding a O antigen biosynthesis gene product that hybridizes under moderately stringent conditions to the DNA of (a) or (b); and related sequences, compositions of matter, vaccines, methods of using, and methods of making.

Подробнее
28-03-2013 дата публикации

Protein C Zymogen and Methods of Use Thereof to Prevent Cancer Metastases

Номер: US20130078302A1
Принадлежит: THE CHILDREN'S HOSPITAL OF PHILADELPHIA

Compositions and methods useful for the inhibition of cancer metastasis are disclosed. 1. A method for treatment or inhibition of metastasis in a cancer patient in need thereof comprising administration of a therapeutically effective amount of a protein C zymogen in a biologically acceptable carrier.2. The method of wherein said protein C zymogen is infused into a patient.3. The method of claim 1 , wherein said protein C zymogen is administered to said patient in an AAV vector comprising protein C zymogen encoding nucleic acid.4. The method of claim 1 , wherein said protein C zymogen is a variant that is not activated in vivo and said cancer is selected from the group consisting of lung cancer claim 1 , pancreatic cancer claim 1 , breast cancer claim 1 , colon cancer claim 1 , melanoma and esophageal cancer.5. The method of claim 1 , comprising administration of at least one chemotherapeutic agent.6. The method of claim 4 , wherein said variant is APC-L38D.7. The method of wherein said variant comprises a S195A substitution.8. The method of claim 4 , wherein said variant comprises a R15Q substitution.9. The method of claim 4 , wherein said variant comprises one claim 4 , two or three of the amino acid substitutions selected from the group consisting of a S195A claim 4 , L38D and R15Q substitution.10. The method of claim 9 , wherein said variant is administered intravenously at least once a day at a dosage between about 10 and 500 μg/kg.11. The method of claim 10 , wherein said variant is administered intravenously at least once a day at a dosage between about 10 and 250 μg/kg.12. The method of claim 9 , wherein said variant is encapsulated in a liposome or mixed with phospholipids or micelles.13. A recombinant AAV vector comprising a nucleic acid encoding a protein C zymogen variant having one claim 9 , two or three amino acid substitutions selected from the group consisting of APC-L38D claim 9 , S195A or R15Q in a pharmaceutically acceptable carrier.14. The method ...

Подробнее