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

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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Autores principales: 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
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2019
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Science
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Acceso en línea:https://doaj.org/article/c5d6b54327424a9fb828402310c7a87f
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https://doaj.org/article/c5d6b54327424a9fb828402310c7a87f

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