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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Nature Publishing Group UK
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6981188/ https://www.ncbi.nlm.nih.gov/pubmed/31980606 http://dx.doi.org/10.1038/s41467-019-14181-3 |