Research ArticleMuscular Dystrophy

Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy

See allHide authors and affiliations

Science Translational Medicine  25 Mar 2020:
Vol. 12, Issue 536, eaay0271
DOI: 10.1126/scitranslmed.aay0271

Article Information

vol. 12 no. 536

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication June 28, 2019
  • Resubmitted December 23, 2019
  • Accepted for publication March 3, 2020
  • .

Author Information

  1. Angela Lek1,2,3,*,
  2. Yuanfan Zhang2,3,
  3. Keryn G. Woodman1,
  4. Shushu Huang1,4,5,
  5. Alec M. DeSimone1,6,
  6. Justin Cohen1,
  7. Vincent Ho1,
  8. James Conner2,
  9. Lillian Mead2,
  10. Andrew Kodani2,3,
  11. Anna Pakula2,3,
  12. Neville Sanjana7,8,
  13. Oliver D. King6,
  14. Peter L. Jones9,
  15. Kathryn R. Wagner10,11,
  16. Monkol Lek1 and
  17. Louis M. Kunkel2,3,12,13,*
  1. 1Department of Genetics, Yale School of Medicine, New Haven, CT 06510, USA.
  2. 2Division of Genetics and Genomics, Boston Children’s Hospital, Boston, MA 02115, USA.
  3. 3Department of Pediatrics and Genetics, Harvard Medical School, Boston, MA 02115, USA.
  4. 4First Affiliated Hospital, Nanjing Medical University, Nanjing 210029, China.
  5. 5Affiliated Hospital of Nantong University, Nantong 226001, China.
  6. 6Wellstone Muscular Dystrophy Program, Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA.
  7. 7New York Genome Center, New York, NY 10013, USA.
  8. 8Department of Biology, New York University, New York, NY 10003, USA.
  9. 9Department of Pharmacology, University of Nevada, Reno School of Medicine, Reno, NV 89557, USA.
  10. 10Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, MD 21205, USA.
  11. 11Departments of Neurology and Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
  12. 12Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  13. 13Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  1. *Corresponding author. Email: kunkel{at}enders.tch.harvard.edu (L.M.K.); angela.lek{at}yale.edu (A.L.)

Altmetric

Article usage

Article usage: March 2020 to May 2020

AbstractFullPdf
Mar 20204888425322
Apr 20201032229211
May 20203256262

Stay Connected to Science Translational Medicine