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A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation

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Science Advances  19 Aug 2020:
Vol. 6, no. 34, eaba5614
DOI: 10.1126/sciadv.aba5614

Article Information

vol. 6 no. 34

PubMed: 
Online ISSN: 
History: 
  • Received for publication December 13, 2019
  • Accepted for publication July 8, 2020
  • .

Author Information

  1. Sybille Böhm1,2,*,
  2. Victoria Splith1,2,*,
  3. Lisa Maria Riedmayr1,2,
  4. René Dominik Rötzer1,2,
  5. Gilles Gasparoni3,
  6. Karl J. V. Nordström3,
  7. Johanna Elisabeth Wagner1,2,
  8. Klara Sonnie Hinrichsmeyer1,2,
  9. Jörn Walter3,
  10. Christian Wahl-Schott4,
  11. Stefanie Fenske1,2,
  12. Martin Biel1,2,
  13. Stylianos Michalakis1,2,5 and
  14. Elvir Becirovic1,2,
  1. 1Center for Integrated Protein Science Munich CIPSM, Munich, Germany.
  2. 2Department of Pharmacy - Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.
  3. 3Department of Genetics, Saarland University, Saarbrücken, Germany.
  4. 4Institute for Neurophysiology, Hannover Medical School (MHH), Hanover, Germany.
  5. 5Department of Ophthalmology, Ludwig-Maximilians-Universität München, Munich, Germany.
  1. Corresponding author. Email: elvir.becirovic{at}cup.uni-muenchen.de
  • * These authors contributed equally to this work.

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