Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
Replacing mutant genes with wildtype copies using adeno-associated virus (AAV) has been explored for the treatment of inherited retinopathies, but the low cargo limit restricts its use. Here the authors describe a single AAV platform that allows local replacement of a mutated sequence with its wildt...
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Autores principales: | Koji M. Nishiguchi, Kosuke Fujita, Fuyuki Miya, Shota Katayama, Toru Nakazawa |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Nature Portfolio
2020
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Acceso en línea: | https://doaj.org/article/cf43721ae08c4135a4828716cd4ce36c |
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