Next-generation muscle-directed gene therapy by in silico vector design

Adeno-associated viral vectors (AAV) are being developed for gene therapy of skeletal muscle, but it is a challenge to achieve robust gene expression. Here, the authors identify muscle-specific cisregulatory elements that lead to a substantial increase in micro-dystrophin and follistatin expression,...

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Main Authors:	S. Sarcar, W. Tulalamba, M. Y. Rincon, J. Tipanee, H. Q. Pham, H. Evens, D. Boon, E. Samara-Kuko, M. Keyaerts, M. Loperfido, E. Berardi, S. Jarmin, P. In’t Veld, G. Dickson, T. Lahoutte, M. Sampaolesi, P. De Bleser, T. VandenDriessche, M. K. Chuah
Format:	article
Language:	EN
Published:	Nature Portfolio 2019
Subjects:	Science Q
Online Access:	https://doaj.org/article/c5d6b54327424a9fb828402310c7a87f
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spelling	oai:doaj.org-article:c5d6b54327424a9fb828402310c7a87f2021-12-02T14:38:58ZNext-generation muscle-directed gene therapy by in silico vector design10.1038/s41467-018-08283-72041-1723https://doaj.org/article/c5d6b54327424a9fb828402310c7a87f2019-01-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-08283-7https://doaj.org/toc/2041-1723Adeno-associated viral vectors (AAV) are being developed for gene therapy of skeletal muscle, but it is a challenge to achieve robust gene expression. Here, the authors identify muscle-specific cisregulatory elements that lead to a substantial increase in micro-dystrophin and follistatin expression, resulting in a safe and sustainable rescue of the dystrophic phenotype in mouse models.S. SarcarW. TulalambaM. Y. RinconJ. TipaneeH. Q. PhamH. EvensD. BoonE. Samara-KukoM. KeyaertsM. LoperfidoE. BerardiS. JarminP. In’t VeldG. DicksonT. LahoutteM. SampaolesiP. De BleserT. VandenDriesscheM. K. ChuahNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-16 (2019)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q S. Sarcar W. Tulalamba M. Y. Rincon J. Tipanee H. Q. Pham H. Evens D. Boon E. Samara-Kuko M. Keyaerts M. Loperfido E. Berardi S. Jarmin P. In’t Veld G. Dickson T. Lahoutte M. Sampaolesi P. De Bleser T. VandenDriessche M. K. Chuah Next-generation muscle-directed gene therapy by in silico vector design
description	Adeno-associated viral vectors (AAV) are being developed for gene therapy of skeletal muscle, but it is a challenge to achieve robust gene expression. Here, the authors identify muscle-specific cisregulatory elements that lead to a substantial increase in micro-dystrophin and follistatin expression, resulting in a safe and sustainable rescue of the dystrophic phenotype in mouse models.
format	article
author	S. Sarcar W. Tulalamba M. Y. Rincon J. Tipanee H. Q. Pham H. Evens D. Boon E. Samara-Kuko M. Keyaerts M. Loperfido E. Berardi S. Jarmin P. In’t Veld G. Dickson T. Lahoutte M. Sampaolesi P. De Bleser T. VandenDriessche M. K. Chuah
author_facet	S. Sarcar W. Tulalamba M. Y. Rincon J. Tipanee H. Q. Pham H. Evens D. Boon E. Samara-Kuko M. Keyaerts M. Loperfido E. Berardi S. Jarmin P. In’t Veld G. Dickson T. Lahoutte M. Sampaolesi P. De Bleser T. VandenDriessche M. K. Chuah
author_sort	S. Sarcar
title	Next-generation muscle-directed gene therapy by in silico vector design
title_short	Next-generation muscle-directed gene therapy by in silico vector design
title_full	Next-generation muscle-directed gene therapy by in silico vector design
title_fullStr	Next-generation muscle-directed gene therapy by in silico vector design
title_full_unstemmed	Next-generation muscle-directed gene therapy by in silico vector design
title_sort	next-generation muscle-directed gene therapy by in silico vector design
publisher	Nature Portfolio
publishDate	2019
url	https://doaj.org/article/c5d6b54327424a9fb828402310c7a87f
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Next-generation muscle-directed gene therapy by in silico vector design

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