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Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

One of the major goals of in vivo gene transfer is to achieve long-term expression of therapeutic transgenes in terminally differentiated cells. The extensive clinical experience and the recent approval of Luxturna(®) (Spark Therapeutics, now Roche) and Zolgensma(®) (AveXis, now Novartis) place vect...

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Detalles Bibliográficos
Autores principales: Gross, David-Alexandre, Tedesco, Novella, Leborgne, Christian, Ronzitti, Giuseppe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9022790/
https://www.ncbi.nlm.nih.gov/pubmed/35464422
http://dx.doi.org/10.3389/fimmu.2022.857276