Efficient precise in vivo base editing in adult dystrophic mice

Base editing is one approach used to correct mutations causing cause Duchenne muscular dystrophy (DMD), but limitations are in the requirement for a specific PAM motif and the large size beyond the packaging capacity of adeno-associated virus (AAV). Here, the authors modify the NG-targeting adenine...

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Autores principales:	Li Xu, Chen Zhang, Haiwen Li, Peipei Wang, Yandi Gao, Nahush A. Mokadam, Jianjie Ma, W. David Arnold, Renzhi Han
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/e267fd77833f4da08de183661fa8161a
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spelling	oai:doaj.org-article:e267fd77833f4da08de183661fa8161a2021-12-02T17:40:23ZEfficient precise in vivo base editing in adult dystrophic mice10.1038/s41467-021-23996-y2041-1723https://doaj.org/article/e267fd77833f4da08de183661fa8161a2021-06-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-23996-yhttps://doaj.org/toc/2041-1723Base editing is one approach used to correct mutations causing cause Duchenne muscular dystrophy (DMD), but limitations are in the requirement for a specific PAM motif and the large size beyond the packaging capacity of adeno-associated virus (AAV). Here, the authors modify the NG-targeting adenine base editor to recognize a broader PAM, devise an intein split strategy to package the otherwise oversized adenine base editor into AAV, and show it efficiently restores dystrophin expression in muscle and heart when systemically injected in a mouse model of DMDLi XuChen ZhangHaiwen LiPeipei WangYandi GaoNahush A. MokadamJianjie MaW. David ArnoldRenzhi HanNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-14 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Li Xu Chen Zhang Haiwen Li Peipei Wang Yandi Gao Nahush A. Mokadam Jianjie Ma W. David Arnold Renzhi Han Efficient precise in vivo base editing in adult dystrophic mice
description	Base editing is one approach used to correct mutations causing cause Duchenne muscular dystrophy (DMD), but limitations are in the requirement for a specific PAM motif and the large size beyond the packaging capacity of adeno-associated virus (AAV). Here, the authors modify the NG-targeting adenine base editor to recognize a broader PAM, devise an intein split strategy to package the otherwise oversized adenine base editor into AAV, and show it efficiently restores dystrophin expression in muscle and heart when systemically injected in a mouse model of DMD
format	article
author	Li Xu Chen Zhang Haiwen Li Peipei Wang Yandi Gao Nahush A. Mokadam Jianjie Ma W. David Arnold Renzhi Han
author_facet	Li Xu Chen Zhang Haiwen Li Peipei Wang Yandi Gao Nahush A. Mokadam Jianjie Ma W. David Arnold Renzhi Han
author_sort	Li Xu
title	Efficient precise in vivo base editing in adult dystrophic mice
title_short	Efficient precise in vivo base editing in adult dystrophic mice
title_full	Efficient precise in vivo base editing in adult dystrophic mice
title_fullStr	Efficient precise in vivo base editing in adult dystrophic mice
title_full_unstemmed	Efficient precise in vivo base editing in adult dystrophic mice
title_sort	efficient precise in vivo base editing in adult dystrophic mice
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/e267fd77833f4da08de183661fa8161a
work_keys_str_mv	AT lixu efficientpreciseinvivobaseeditinginadultdystrophicmice AT chenzhang efficientpreciseinvivobaseeditinginadultdystrophicmice AT haiwenli efficientpreciseinvivobaseeditinginadultdystrophicmice AT peipeiwang efficientpreciseinvivobaseeditinginadultdystrophicmice AT yandigao efficientpreciseinvivobaseeditinginadultdystrophicmice AT nahushamokadam efficientpreciseinvivobaseeditinginadultdystrophicmice AT jianjiema efficientpreciseinvivobaseeditinginadultdystrophicmice AT wdavidarnold efficientpreciseinvivobaseeditinginadultdystrophicmice AT renzhihan efficientpreciseinvivobaseeditinginadultdystrophicmice
_version_	1718379796482752512

Efficient precise in vivo base editing in adult dystrophic mice

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