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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Main Authors:	Li Xu, Chen Zhang, Haiwen Li, Peipei Wang, Yandi Gao, Nahush A. Mokadam, Jianjie Ma, W. David Arnold, Renzhi Han
Format:	Article
Language:	English
Published:	Nature Publishing Group 2021-06-01
Series:	Nature Communications
Online Access:	https://doi.org/10.1038/s41467-021-23996-y

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spelling	doaj-e267fd77833f4da08de183661fa8161a2021-06-20T11:13:05ZengNature Publishing GroupNature Communications2041-17232021-06-0112111410.1038/s41467-021-23996-yEfficient precise in vivo base editing in adult dystrophic miceLi Xu0Chen Zhang1Haiwen Li2Peipei Wang3Yandi Gao4Nahush A. Mokadam5Jianjie Ma6W. David Arnold7Renzhi Han8Division of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterDepartment of Neurology, The Ohio State University Wexner Medical CenterDivision of Cardiac Surgery, Department of Surgery, The Ohio State University Wexner Medical CenterBase 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 DMDhttps://doi.org/10.1038/s41467-021-23996-y
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Li Xu Chen Zhang Haiwen Li Peipei Wang Yandi Gao Nahush A. Mokadam Jianjie Ma W. David Arnold Renzhi Han
spellingShingle	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 Nature Communications
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 Publishing Group
series	Nature Communications
issn	2041-1723
publishDate	2021-06-01
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
url	https://doi.org/10.1038/s41467-021-23996-y
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Efficient precise in vivo base editing in adult dystrophic mice

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