A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

Efforts to develop gene therapies for hearing loss have been hampered by the lack of safe, efficient, and clinically relevant delivery modalities(1, 2). Here we demonstrate the safety and efficiency of Anc80L65, a rationally designed synthetic vector(3), for transgene delivery to the mouse cochlea....

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Detalles Bibliográficos
Autores principales: Landegger, Lukas D., Pan, Bifeng, Askew, Charles, Wassmer, Sarah, Gluck, Sarah, Galvin, Alice, Taylor, Ruth, Forge, Andrew, Stankovic, Konstantina M., Holt, Jeffrey R., Vandenberghe, Luk H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5340646/
https://www.ncbi.nlm.nih.gov/pubmed/28165475
http://dx.doi.org/10.1038/nbt.3781

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5340646/
https://www.ncbi.nlm.nih.gov/pubmed/28165475
http://dx.doi.org/10.1038/nbt.3781

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