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Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
Supplementing wildtype copies of functionally defective genes with adeno-associated virus (AAV) is a strategy being explored clinically for various retinal dystrophies. However, the low cargo limit of this vector allows its use in only a fraction of patients with mutations in relatively small pathog...
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| Publié dans: | Nat Commun |
|---|---|
| Auteurs principaux: | , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
Nature Publishing Group UK
2020
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6981188/ https://ncbi.nlm.nih.gov/pubmed/31980606 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-019-14181-3 |
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