Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease

Wang et al. describe the development of a 3D in vitro model of human skeletal muscle that recapitulates the pathophysiology of Pompe disease. They further define a Pompe disease-specific transcriptional signature, confirm its presence in the Pompe myobundles, and detect a partial reversal of the sig...

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Autores principales:	Jason Wang, Chris J. Zhou, Alastair Khodabukus, Sabrina Tran, Sang-Oh Han, Aaron L. Carlson, Lauran Madden, Priya S. Kishnani, Dwight D. Koeberl, Nenad Bursac
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Biology (General) QH301-705.5
Acceso en línea:	https://doaj.org/article/e7bed5b1c04b4acdbdebddf5309410f2
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spelling	oai:doaj.org-article:e7bed5b1c04b4acdbdebddf5309410f22021-12-02T14:29:14ZThree-dimensional tissue-engineered human skeletal muscle model of Pompe disease10.1038/s42003-021-02059-42399-3642https://doaj.org/article/e7bed5b1c04b4acdbdebddf5309410f22021-05-01T00:00:00Zhttps://doi.org/10.1038/s42003-021-02059-4https://doaj.org/toc/2399-3642Wang et al. describe the development of a 3D in vitro model of human skeletal muscle that recapitulates the pathophysiology of Pompe disease. They further define a Pompe disease-specific transcriptional signature, confirm its presence in the Pompe myobundles, and detect a partial reversal of the signature upon in vitro treatment of myobundles with rhGAA.Jason WangChris J. ZhouAlastair KhodabukusSabrina TranSang-Oh HanAaron L. CarlsonLauran MaddenPriya S. KishnaniDwight D. KoeberlNenad BursacNature PortfolioarticleBiology (General)QH301-705.5ENCommunications Biology, Vol 4, Iss 1, Pp 1-14 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Biology (General) QH301-705.5
spellingShingle	Biology (General) QH301-705.5 Jason Wang Chris J. Zhou Alastair Khodabukus Sabrina Tran Sang-Oh Han Aaron L. Carlson Lauran Madden Priya S. Kishnani Dwight D. Koeberl Nenad Bursac Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
description	Wang et al. describe the development of a 3D in vitro model of human skeletal muscle that recapitulates the pathophysiology of Pompe disease. They further define a Pompe disease-specific transcriptional signature, confirm its presence in the Pompe myobundles, and detect a partial reversal of the signature upon in vitro treatment of myobundles with rhGAA.
format	article
author	Jason Wang Chris J. Zhou Alastair Khodabukus Sabrina Tran Sang-Oh Han Aaron L. Carlson Lauran Madden Priya S. Kishnani Dwight D. Koeberl Nenad Bursac
author_facet	Jason Wang Chris J. Zhou Alastair Khodabukus Sabrina Tran Sang-Oh Han Aaron L. Carlson Lauran Madden Priya S. Kishnani Dwight D. Koeberl Nenad Bursac
author_sort	Jason Wang
title	Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
title_short	Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
title_full	Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
title_fullStr	Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
title_full_unstemmed	Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
title_sort	three-dimensional tissue-engineered human skeletal muscle model of pompe disease
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/e7bed5b1c04b4acdbdebddf5309410f2
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Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease

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