Isolated modified AAV9 capsid protein VP1
OwnerAnna Nikolaevna Strelkova, Tatiana Evgenievna Shugaeva +3inventors
InventorsAnna Nikolaevna Strelkova, Tatiana Evgenievna Shugaeva, Pavel Mikhailovich Gershovich, Pavel Andreevich Iakovlev, Dmitry Valentinovich Morozov
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Abstract
The present application relates to the fields of gene therapy and molecular biology. More specifically, the present invention relates to an isolated modified capsid protein VP1 from adeno-associated virus serotype 9 (AAV9) comprising one or more amino acid substitutions compared to the wild-type AAV9 capsid protein VP1, which substitutions increase the efficiency of production (assembly) of the vector based on recombinant adeno-associated virus serotype 9 (rAAV9), to a capsid and a vector based on the above VP1, as well as to uses thereof.
Claims21
An isolated modified capsid protein VP1 from adeno-associated virus serotype 9 (AAV9) for production of encapsidated viral vectors based on recombinant adeno-associated virus serotype 9 (rAAV9), said isolated modified capsid protein VP1 comprising a wild-type AAV9 capsid protein VP1 amino acid sequence encoded by the Cap gene with one or more substitutions that are selected from the group consisting of:
- F422W,
- F422W and Y446F, and
- I601M,
- wherein the wild-type AAV9 capsid protein VP1 amino acid sequence has the amino acid sequence represented by SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.