公开(公告)号：US20250127923A1

公开(公告)日：2025-04-24

申请号：US18684921

申请日：2022-08-21

Applicant: JOINT STOCK COMPANY "BIOCAD"

Inventor： Anna Nikolaevna STRELKOVA ,  Sergei Aleksandrovich LEGOTSKII ,  Tatiana Evgenievna SHUGAEVA ,  Pavel Mikhailovich GERSHOVICH ,  Alexander Vladimirovich PROKOFYEV ,  Mariya Pavlovna PEREPELKINA ,  Pavel Andreevich IAKOVLEV ,  Dmitry Valentinovich MOROZOV

IPC: A61K48/00 ,  C07K14/005 ,  C12N15/86

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 5 (AAV5), comprising one or more amino acid substitutions compared to the wild-type AAV5 capsid protein VP1, which substitutions increase the efficiency of transduction, increase the efficiency of packaging of AAV viral genomes with rAAV5-based products, and also increase the efficiency of production (assembly) of a vector based on recombinant adeno-associated virus serotype 5 (rAAV5), to a capsid and a vector based on the above VP1, as well as to uses thereof.