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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Autor*in:	Sourav K. Bose [verfasserIn] Brandon M. White [verfasserIn] Meghana V. Kashyap [verfasserIn] Apeksha Dave [verfasserIn] Felix R. De Bie [verfasserIn] Haiying Li [verfasserIn] Kshitiz Singh [verfasserIn] Pallavi Menon [verfasserIn] Tiankun Wang [verfasserIn] Shiva Teerdhala [verfasserIn] Vishal Swaminathan [verfasserIn] Heather A. Hartman [verfasserIn] Sowmya Jayachandran [verfasserIn] Prashant Chandrasekaran [verfasserIn] Kiran Musunuru [verfasserIn] Rajan Jain [verfasserIn] David B. Frank [verfasserIn] Philip Zoltick [verfasserIn] William H. Peranteau [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2021

Übergeordnetes Werk:	In: Nature Communications - Nature Portfolio, 2016, 12(2021), 1, Seite 16
Übergeordnetes Werk:	volume:12 ; year:2021 ; number:1 ; pages:16

Links:	Link aufrufen Link aufrufen Link aufrufen Journal toc

DOI / URN:	10.1038/s41467-021-24443-8

Katalog-ID:	DOAJ066755158

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author	Sourav K. Bose
spellingShingle	Sourav K. Bose misc Science misc Q In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
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topic_title	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
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title	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
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author_browse	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
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title_sort	in utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
title_auth	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
abstract	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.
abstractGer	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.
abstract_unstemmed	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.
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title_short	In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
url	https://doi.org/10.1038/s41467-021-24443-8 https://doaj.org/article/a932a6ec2ba7497281b8dba976e50d99 https://doaj.org/toc/2041-1723
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