Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1

Myotonic dystrophy type 1 (DM1) is associated with aberrant transcript splicing. Here, the authors develop a transgenic mouse model expressing a bi-chromatic reporter system that allows non-invasive monitoring of splicing of a transcript altered in DM1 in vivo, and show that it allows for evaluation...

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Autores principales:	Ningyan Hu, Layal Antoury, Timothy M. Baran, Soumya Mitra, C. Frank Bennett, Frank Rigo, Thomas H. Foster, Thurman M. Wheeler
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2018
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/b1565fb81d1d487aac6b620c6b16e1b3
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spelling	oai:doaj.org-article:b1565fb81d1d487aac6b620c6b16e1b32021-12-02T17:33:17ZNon-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 110.1038/s41467-018-07517-y2041-1723https://doaj.org/article/b1565fb81d1d487aac6b620c6b16e1b32018-12-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-07517-yhttps://doaj.org/toc/2041-1723Myotonic dystrophy type 1 (DM1) is associated with aberrant transcript splicing. Here, the authors develop a transgenic mouse model expressing a bi-chromatic reporter system that allows non-invasive monitoring of splicing of a transcript altered in DM1 in vivo, and show that it allows for evaluation of the therapeutic response to treatment with antisense oligonucleotides.Ningyan HuLayal AntouryTimothy M. BaranSoumya MitraC. Frank BennettFrank RigoThomas H. FosterThurman M. WheelerNature PortfolioarticleScienceQENNature Communications, Vol 9, Iss 1, Pp 1-15 (2018)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Ningyan Hu Layal Antoury Timothy M. Baran Soumya Mitra C. Frank Bennett Frank Rigo Thomas H. Foster Thurman M. Wheeler Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
description	Myotonic dystrophy type 1 (DM1) is associated with aberrant transcript splicing. Here, the authors develop a transgenic mouse model expressing a bi-chromatic reporter system that allows non-invasive monitoring of splicing of a transcript altered in DM1 in vivo, and show that it allows for evaluation of the therapeutic response to treatment with antisense oligonucleotides.
format	article
author	Ningyan Hu Layal Antoury Timothy M. Baran Soumya Mitra C. Frank Bennett Frank Rigo Thomas H. Foster Thurman M. Wheeler
author_facet	Ningyan Hu Layal Antoury Timothy M. Baran Soumya Mitra C. Frank Bennett Frank Rigo Thomas H. Foster Thurman M. Wheeler
author_sort	Ningyan Hu
title	Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
title_short	Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
title_full	Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
title_fullStr	Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
title_full_unstemmed	Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
title_sort	non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
publisher	Nature Portfolio
publishDate	2018
url	https://doaj.org/article/b1565fb81d1d487aac6b620c6b16e1b3
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Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1

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