Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism

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专利权人:

Association Institut De Myologie;Inserm (Institut national de la santé et de la recherche médicale);Universite D'evry Val D'essonne;Sorbonne Universite;Genethon

发明人:

RICHARD, Isabelle,GICQUEL, Evelyne,MINGOZZI, Frederico,RONZITTI, Giuseppe,VIDAL, Patrice

申请号：

AU2019249890

公开号：

AU2019249890A1

申请日:

2019.04.04

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.

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中国工程科技知识中心

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http://www.ckcest.cn/home/