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RAAV WITH CHEMICALLY MODIFIED CAPSID

Abstract : 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.
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https://www.hal.inserm.fr/inserm-02874921
Contributor : Nicolas Jaulin <>
Submitted on : Friday, June 19, 2020 - 12:01:30 PM
Last modification on : Wednesday, June 24, 2020 - 10:06:30 AM

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  • HAL Id : inserm-02874921, version 1

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Citation

Mathieu Mével, David Deniaud, Eduard Ayuso. RAAV WITH CHEMICALLY MODIFIED CAPSID. France, Patent n° : WO2017EP64089 20170609. 2019. ⟨inserm-02874921⟩

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