FIELD: biotechnology.
SUBSTANCE: described is a recombinant adeno-associated virus (AAV) vector comprising (a) variant AAV2 capsid protein, wherein the variant AAV2 capsid protein comprises at least four amino acid substitutions compared to the non-mutant type AAV2 capsid protein; where at least four amino acid substitutions are present in following positions in AAV2 sequence of capsid protein: 457, 492, 499 and 533; and (b) a heterologous nucleic acid containing a nucleotide sequence encoding a gene product.
EFFECT: invention can be used for treating neurological and eye diseases, as well as liver.
17 cl, 39 dwg, 1 tbl, 6 ex
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Year |
Author |
Number |
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2017 |
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2016 |
|
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2012 |
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|
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2017 |
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|
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|
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2015 |
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|
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|
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2018 |
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|
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|
RU2811445C2 |
