Research ArticleHEALTH AND MEDICINE

A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency

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Science Advances  12 Feb 2020:
Vol. 6, no. 7, eaax5701
DOI: 10.1126/sciadv.aax5701

Article Information

vol. 6 no. 7

Online ISSN: 
History: 
  • Received for publication April 3, 2019
  • Accepted for publication September 30, 2019
  • .

Author Information

  1. Lili Wang1,*,
  2. Yang Yang1,2,*,
  3. Camilo Breton1,
  4. Peter Bell1,
  5. Mingyao Li3,
  6. Jia Zhang1,,
  7. Yan Che1,
  8. Alexei Saveliev1,
  9. Zhenning He1,
  10. John White1,
  11. Caitlin Latshaw1,
  12. Chenyu Xu4,
  13. Deirdre McMenamin1,
  14. Hongwei Yu1,
  15. Hiroki Morizono4,
  16. Mark L. Batshaw4 and
  17. James M. Wilson1,
  1. 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  2. 2State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University, and Collaborative Innovation Center for Biotherapy, Chengdu, Sichuan, China.
  3. 3Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, PA 19104, USA.
  4. 4Center for Genetic Medicine Research, Children’s Research Institute, Children’s National Hospital, Washington, DC 20010, USA.
  1. Corresponding author. Email: wilsonjm{at}upenn.edu
  • * These authors contributed equally to this work.

  • Present address: Sarepta Therapeutics, Burlington, MA, USA.

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