A Muscle Hybrid Promoter as a Novel Tool for Gene Therapy

Gene therapy is a promising strategy to cure rare diseases. The lack of regulatory sequences ensuring specific and robust expression in skeletal and cardiac muscle is a substantial limitation of gene therapy efficiency targeting the muscle tissue. Here we describe a novel muscle hybrid (MH) promoter...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Piekarowicz, Katarzyna, Bertrand, Anne T., Azibani, Feriel, Beuvin, Maud, Julien, Laura, Machowska, Magdalena, Bonne, Gisèle, Rzepecki, Ryszard
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6807297/
https://ncbi.nlm.nih.gov/pubmed/31660418
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.09.001
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