Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

Gene therapy of Usher syndrome type 1B (USH1B) due to mutations in the large Myosin VIIA (MYO7A) gene is limited by the packaging capacity of adeno-associated viral (AAV) vectors. To overcome this, we have previously developed dual AAV8 vectors which encode human MYO7A (dual AAV8.MYO7A). Here we sho...

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Detalles Bibliográficos
Autores principales: Ferla, Rita, Dell’Aquila, Fabio, Doria, Monica, Ferraiuolo, Maria, Noto, Alessia, Grazioli, Fabiana, Ammendola, Virginia, Testa, Francesco, Melillo, Paolo, Iodice, Carolina, Risca, Giulia, Tedesco, Novella, le Brun, Pierre Romain, Surace, Enrico Maria, Simonelli, Francesca, Galimberti, Stefania, Valsecchi, Maria Grazia, Marteau, Jean-Brice, Veron, Philippe, Colloca, Stefano, Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9996380/
https://www.ncbi.nlm.nih.gov/pubmed/36910588
http://dx.doi.org/10.1016/j.omtm.2023.02.002

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