In vivo genome editing of the albumin locus as a platform for protein replacement therapy.

@article{Sharma2015InVG,
  title={In vivo genome editing of the albumin locus as a platform for protein replacement therapy.},
  author={Rajiv Sharma and Xavier M Anguela and Yannick Doyon and Thomas Wechsler and Russell C. DeKelver and Scott Sproul and David E. Paschon and Jeffrey C. Miller and Robert J Davidson and David A. Shivak and Shangzhen Zhou and Julianne M Rieders and Philip D. Gregory and Michael C. Holmes and Edward J. Rebar and K. A. High},
  journal={Blood},
  year={2015},
  volume={126 15},
  pages={
          1777-84
        }
}
Site-specific genome editing provides a promising approach for achieving long-term, stable therapeutic gene expression. Genome editing has been successfully applied in a variety of preclinical models, generally focused on targeting the diseased locus itself; however, limited targeting efficiency or insufficient expression from the endogenous promoter may impede the translation of these approaches, particularly if the desired editing event does not confer a selective growth advantage. Here we… CONTINUE READING
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