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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Auteurs principaux: Koji M. Nishiguchi, Kosuke Fujita, Fuyuki Miya, Shota Katayama, Toru Nakazawa
Format: article
Langue:EN
Publié: Nature Portfolio 2020
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Accès en ligne:https://doaj.org/article/cf43721ae08c4135a4828716cd4ce36c
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