07-05-2015 дата публикации
Номер: US20150126588A1
Принадлежит:
Disclosed herein are adeno associated viral plasmids and viral vectors. Also disclosed are methods of using adeno associated viral vectors. 1. An adeno associated virus plasmid comprising:a polynucleotide that encodes a protein with the amino acid sequence of SEQ ID NO: 1, or a fragment thereof, further comprising a mutation at at least one of L380, T381, L382, N383, I440, D441, Y446, L447, T450, I451, V465, P468, S469, N470, M471, Q474, G475, Y484, R485, E500, F501, W503, P504, G505, R514, N515, S516, L517, and Q590.2. The adeno associated virus plasmid of claim 1 , wherein the mutation is at at least one of I451 claim 1 , V465 claim 1 , P468 claim 1 , S469 claim 1 , N470 claim 1 , M471 claim 1 , G475 claim 1 , Y484 claim 1 , E500 claim 1 , F501 claim 1 , N515 claim 1 , and L517.3. The adeno associated virus plasmid of claim 1 , wherein the mutation is at least one of Y484A and R485A.4. The adeno associated virus plasmid of claim 1 , wherein the mutation is in a viral capsid protein.5. An adeno associated virus plasmid comprising:a polynucleotide that encodes a protein with the amino acid sequence of SEQ ID NO: 4, or a fragment thereof, further comprising a mutation at at least one of Q464, A467, D469, I470, R471, D472, S474, Y500, S501, and D514.6. The adeno associated virus plasmid of claim 5 , wherein the mutation is at least one of Q464R claim 5 , Q464V claim 5 , A467P claim 5 , D469G claim 5 , D469T claim 5 , I470M claim 5 , R471A claim 5 , R471S claim 5 , D472V claim 5 , D472E claim 5 , D472N claim 5 , S474P claim 5 , S474A claim 5 , S474G claim 5 , Y500E claim 5 , S501F claim 5 , and D514R.7. The plasmid of claim 6 , wherein the polynucleotide encodes a protein with an amino acid sequence selected from at least one of SEQ ID NO: 5 claim 6 , SEQ ID NO: 6 claim 6 , SEQ ID NO: 7 claim 6 , SEQ ID NO: 8 claim 6 , SEQ ID NO: 9 claim 6 , SEQ ID NO: 10 claim 6 , SEQ ID NO: 11 claim 6 , SEQ ID NO: 12 claim 6 , SEQ ID NO: 13 claim 6 , SEQ ID NO: 14 claim 6 , SEQ ID NO ...
Подробнее