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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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Bibliografski detalji
Izdano u:Nat Biotechnol
Glavni autori: 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.
Format: Artigo
Jezik:Inglês
Izdano: 2017
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5340646/
https://ncbi.nlm.nih.gov/pubmed/28165475
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nbt.3781
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