CENTRE HOSPITALIER UNIVERSITAIRE DE NANTES;CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS);INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM);UNIVERSITE DE NANTES
发明人:
Mathieu MEVEL,David DENIAUD,Eduard AYUSO
申请号：
US16308740
公开号：
US20190388557A1
申请日:
2017.06.09
申请国别(地区):
US
年份:
2019
代理人:
摘要：
The invention is directed to the field of gene therapy, i.e. gene delivery into target cells, tissue, organ and organism, and more particularly to gene delivery via viral vectors. The inventors showed that it is possible by chemical coupling to modulate the coupling of a ligand in the surface of the capsid of AAV, for example AAV2 and AAV3b. In particular, the present invention relates to a recombinant Adeno-Associated Virus (rAAV) vector particle having at least one primary amino group contained in the capsid proteins, chemically coupled with at least one ligand L, wherein coupling of said ligand L is implemented through a bond comprising a —；CSNH—； bond and an optionally substituted aromatic moiety. Particularly, the inventors tested the chemical coupling of mannose ligand on AAV2 for subretinally injection to rats. The present invention further relates to a method for chemically coupling an Adeno-Associated Virus (AAV) vector particle with at least one ligand L and to a Recombinant Adeno-Associated Virus (rAAV) vector particle obtained by said method as well as a pharmaceutical composition comprising it and their corresponding medical use.
