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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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| Publicado no: | Nat Biotechnol |
|---|---|
| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2017
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| Assuntos: | |
| Acesso em linha: | 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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