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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Detaylı Bibliyografya
Yayımlandı:Nat Biotechnol
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2017
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Article
Online Erişim: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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