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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Bibliographic Details
Main Authors: Katarzyna Piekarowicz, Anne T. Bertrand, Feriel Azibani, Maud Beuvin, Laura Julien, Magdalena Machowska, Gisèle Bonne, Ryszard Rzepecki
Format: Article
Language:English
Published: Elsevier 2019-12-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050119300981