piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder caused by the absence of dystrophin. We developed a novel gene therapy approach based on the use of the piggyBac (PB) transposon system to deliver the coding DNA sequence (CDS) of either full-length human dystrophin (DYS: 11.1 kb)...

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Detalles Bibliográficos
Autores principales: Loperfido, Mariana, Jarmin, Susan, Dastidar, Sumitava, Di Matteo, Mario, Perini, Ilaria, Moore, Marc, Nair, Nisha, Samara-Kuko, Ermira, Athanasopoulos, Takis, Tedesco, Francesco Saverio, Dickson, George, Sampaolesi, Maurilio, VandenDriessche, Thierry, Chuah, Marinee K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2016
Materias:
Molecular Biology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4737162/
https://www.ncbi.nlm.nih.gov/pubmed/26682797
http://dx.doi.org/10.1093/nar/gkv1464

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