In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

Lysosomal storage diseases like mucopolysaccharidosis type I (MPS I) cause pathology before birth and result in early morbidity and mortality. Here, the authors show that in utero base editing mediates multi-organ phenotypic and survival benefits in a mouse model recapitulating a common human MPSI m...

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Autores principales:	Sourav K. Bose, Brandon M. White, Meghana V. Kashyap, Apeksha Dave, Felix R. De Bie, Haiying Li, Kshitiz Singh, Pallavi Menon, Tiankun Wang, Shiva Teerdhala, Vishal Swaminathan, Heather A. Hartman, Sowmya Jayachandran, Prashant Chandrasekaran, Kiran Musunuru, Rajan Jain, David B. Frank, Philip Zoltick, William H. Peranteau
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/a932a6ec2ba7497281b8dba976e50d99
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spelling	oai:doaj.org-article:a932a6ec2ba7497281b8dba976e50d992021-12-02T17:01:29ZIn utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease10.1038/s41467-021-24443-82041-1723https://doaj.org/article/a932a6ec2ba7497281b8dba976e50d992021-07-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-24443-8https://doaj.org/toc/2041-1723Lysosomal storage diseases like mucopolysaccharidosis type I (MPS I) cause pathology before birth and result in early morbidity and mortality. Here, the authors show that in utero base editing mediates multi-organ phenotypic and survival benefits in a mouse model recapitulating a common human MPSI mutation.Sourav K. BoseBrandon M. WhiteMeghana V. KashyapApeksha DaveFelix R. De BieHaiying LiKshitiz SinghPallavi MenonTiankun WangShiva TeerdhalaVishal SwaminathanHeather A. HartmanSowmya JayachandranPrashant ChandrasekaranKiran MusunuruRajan JainDavid B. FrankPhilip ZoltickWilliam H. PeranteauNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-16 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Sourav K. Bose Brandon M. White Meghana V. Kashyap Apeksha Dave Felix R. De Bie Haiying Li Kshitiz Singh Pallavi Menon Tiankun Wang Shiva Teerdhala Vishal Swaminathan Heather A. Hartman Sowmya Jayachandran Prashant Chandrasekaran Kiran Musunuru Rajan Jain David B. Frank Philip Zoltick William H. Peranteau In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
description	Lysosomal storage diseases like mucopolysaccharidosis type I (MPS I) cause pathology before birth and result in early morbidity and mortality. Here, the authors show that in utero base editing mediates multi-organ phenotypic and survival benefits in a mouse model recapitulating a common human MPSI mutation.
format	article
author	Sourav K. Bose Brandon M. White Meghana V. Kashyap Apeksha Dave Felix R. De Bie Haiying Li Kshitiz Singh Pallavi Menon Tiankun Wang Shiva Teerdhala Vishal Swaminathan Heather A. Hartman Sowmya Jayachandran Prashant Chandrasekaran Kiran Musunuru Rajan Jain David B. Frank Philip Zoltick William H. Peranteau
author_facet	Sourav K. Bose Brandon M. White Meghana V. Kashyap Apeksha Dave Felix R. De Bie Haiying Li Kshitiz Singh Pallavi Menon Tiankun Wang Shiva Teerdhala Vishal Swaminathan Heather A. Hartman Sowmya Jayachandran Prashant Chandrasekaran Kiran Musunuru Rajan Jain David B. Frank Philip Zoltick William H. Peranteau
author_sort	Sourav K. Bose
title	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
title_short	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
title_full	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
title_fullStr	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
title_full_unstemmed	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
title_sort	in utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/a932a6ec2ba7497281b8dba976e50d99
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In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

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