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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Nature Portfolio
2019
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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) |
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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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