25-01-2018 дата публикации
Номер: US20180021412A1
Принадлежит:
The present invention relates in part to nucleic acids, including nucleic acids encoding proteins, therapeutics and cosmetics comprising nucleic acids, methods for delivering nucleic acids to cells, tissues, organs, and patients, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, therapeutics, and cosmetics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules, including cells present in vivo. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described. 1202.-. (canceled)203. An in vivo method for treating epidermolysis bullosa , comprisingdelivering a synthetic RNA encoding a gene-editing protein that targets a COL7 gene to a patient in need thereof andinducing a double-strand break in the COL7 gene of the patient's keratinocytes, ["the synthetic RNA is delivered to the patient's keratinocytes by injection to the epidermis and", 'the gene-editing protein comprises a DNA-binding domain and a nuclease domain., 'thereby eliminating a mutation that is at least partially responsible for a disease phenotype, wherein204. The method of claim 203 , wherein the gene-editing protein is capable of targeting a nucleic acid sequence that encodes the amino acid sequence of SEQ ID NO: 78.205. The method of claim 203 , wherein the gene-editing protein is selected from the group consisting of a nuclease claim 203 , a transcription activator-like effector nuclease (TALEN) claim 203 , a zinc-finger nuclease claim 203 , a meganuclease claim 203 , a nickase claim 203 , and a clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein.206. The method of claim 203 , wherein the synthetic RNA further comprises one or both of a 5′-cap structure and a 3′- ...
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