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

There is an urgent need to develop the next-generation vectors for gene therapy of muscle disorders, given the relatively modest advances in clinical trials. These vectors should express substantially higher levels of the therapeutic transgene, enabling the use of lower and safer vector doses. In th...

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Udgivet i:Nat Commun
Main Authors: Sarcar, S., Tulalamba, W., Rincon, M. Y., Tipanee, J., Pham, H. Q., Evens, H., Boon, D., Samara-Kuko, E., Keyaerts, M., Loperfido, M., Berardi, E., Jarmin, S., In’t Veld, P., Dickson, G., Lahoutte, T., Sampaolesi, M., De Bleser, P., VandenDriessche, T., Chuah, M. K.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6353880/
https://ncbi.nlm.nih.gov/pubmed/30700722
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-018-08283-7
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