CRG Scientific Sessions

10/01/202512:00MARIE CURIEPRBB-CRG SessionsAlberto AuricchioTIGEM - Telethon Institute of Genetics and Medicine"ExpEditing AAV gene therapy"Host: Pia CosmaAbstract:In vivo gene therapy with adeno-associated viral (AAV) vectors is holding its promise for treatment of genetic diseases, yet some challenges remain that prevent to expand this approach to a larger patients population. These include: AAV cargo capacity limited to about 5 kb which prevents their application to conditions due to mutations in genes with a larger coding sequence; the episomal status of AAV genomes which results in short-term expression in proliferating tissues; efficient...