Recombinant adeno-associated virus with modified aav capsid polypeptides

The present invention relates to a modified adeno-associated virus (AAV) capsid polypeptide and a novel recombinant adeno-associated virus (rAAV) comprising the modified AAV capsid polypeptide for delivering the gene product for the therapy. The present invention also relates to a pharmaceutical com...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: LI Heping, REN Qiongqiong, WU Bing, SHU Yanqun, HOU Jinzhao, XIAO Lumei, LI Peilu
Format: Patent
Sprache:eng ; heb
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Zusammenfassung:The present invention relates to a modified adeno-associated virus (AAV) capsid polypeptide and a novel recombinant adeno-associated virus (rAAV) comprising the modified AAV capsid polypeptide for delivering the gene product for the therapy. The present invention also relates to a pharmaceutical composition comprising the rAAV of the present invention, and a method of treating a retinal disease comprising the administration of the rAAV or the pharmaceutical composition of the present invention to an eye of a subject in need thereof.