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
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2020
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/cf43721ae08c4135a4828716cd4ce36c
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spelling	oai:doaj.org-article:cf43721ae08c4135a4828716cd4ce36c2021-12-02T15:39:14ZSingle AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice10.1038/s41467-019-14181-32041-1723https://doaj.org/article/cf43721ae08c4135a4828716cd4ce36c2020-01-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-14181-3https://doaj.org/toc/2041-1723Replacing 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 wildtype counterpart, based on combined CRISPR-Cas9 and micro-homology-mediated end joining.Koji M. NishiguchiKosuke FujitaFuyuki MiyaShota KatayamaToru NakazawaNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-9 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Koji M. Nishiguchi Kosuke Fujita Fuyuki Miya Shota Katayama Toru Nakazawa Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
description	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 wildtype counterpart, based on combined CRISPR-Cas9 and micro-homology-mediated end joining.
format	article
author	Koji M. Nishiguchi Kosuke Fujita Fuyuki Miya Shota Katayama Toru Nakazawa
author_facet	Koji M. Nishiguchi Kosuke Fujita Fuyuki Miya Shota Katayama Toru Nakazawa
author_sort	Koji M. Nishiguchi
title	Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
title_short	Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
title_full	Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
title_fullStr	Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
title_full_unstemmed	Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
title_sort	single aav-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/cf43721ae08c4135a4828716cd4ce36c
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Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice

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