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Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts

Duchenne muscular dystrophy (DMD) is a lethal muscle-wasting disease currently without cure. We investigated the use of the PiggyBac transposon for full-length dystrophin expression in murine mesoangioblast (MABs) progenitor cells. DMD murine MABs were transfected with transposable expression vector...

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Veröffentlicht in:Mol Ther
Hauptverfasser: Iyer, Pavithra S., Mavoungou, Lionel O., Ronzoni, Flavio, Zemla, Joanna, Schmid-Siegert, Emanuel, Antonini, Stefania, Neff, Laurence A., Dorchies, Olivier M., Jaconi, Marisa, Lekka, Malgorzata, Messina, Graziella, Mermod, Nicolas
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2018
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6079556/
https://ncbi.nlm.nih.gov/pubmed/29503200
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.01.021
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