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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Detalles Bibliográficos
Autores principales: Nishiguchi, Koji M., Fujita, Kosuke, Miya, Fuyuki, Katayama, Shota, Nakazawa, Toru
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
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

Internet

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

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