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

@inproceedings{Benabdallah2013TargetedGA,
  title={Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation},
  author={Basma Fattouma Benabdallah and Arnaud Duval and Joel Rousseau and Pierre Chapdelaine and Michael C. Holmes and Eli Haddad and Jacques Pierre Tremblay and Christian M. Beaus{\'e}jour},
  booktitle={Molecular therapy. Nucleic acids},
  year={2013}
}
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 fluorescent protein (eGFP) or the microdystrophin genes into human myoblasts. Using integrase-defective lentiviral vectors (IDLVs) and chimeric adenoviral vectors to transiently deliver template DNA… CONTINUE READING
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