Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.

@article{Gori2015DeliveryAS,
  title={Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.},
  author={Jennifer L. Gori and Patrick D. Hsu and Morgan L. Maeder and Shen Shen and G. Grant Welstead and David A. Bumcrot},
  journal={Human gene therapy},
  year={2015},
  volume={26 7},
  pages={
          443-51
        }
}
Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated 9 (Cas9) technology is revolutionizing the study of gene function and likely will give rise to an entire new class of therapeutics for a wide range of diseases. Achieving this goal requires not only characterization of the technology for efficacy and specificity but also optimization of its delivery to the target cells for each disease indication. In this review we survey the various… 
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166 Citations
Highly Influential Citations
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166 Citations

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...

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