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Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation

Zinc finger nucleases (ZFN) can facilitate targeted gene addition to the genome while minimizing the risks of insertional mutagenesis. Here, we used a previously characterized ZFN pair targeting the chemokine (C-C motif) receptor 5 (CCR5) locus to introduce, as a proof of concept, the enhanced green...

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Autori principali: Basma F Benabdallah, Arnaud Duval, Joel Rousseau, Pierre Chapdelaine, Michael C Holmes, Eli Haddad, Jacques P Tremblay, Christian M Beauséjour
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2013-01-01
Serie:Molecular Therapy: Nucleic Acids
Soggetti:
dystrophin
gene addition
gene therapy
myoblast
zinc finger nuclease
Accesso online:http://www.sciencedirect.com/science/article/pii/S2162253116301238
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