Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases.

Thomas Kocher (First author), Patricia Peking (Co-author), Alfred Klausegger (Co-author), Eva Maria Murauer (Co-author), Josefina Piñón Hofbauer (Co-author), Verena Wally (Co-author), Thomas Lettner (Co-author), Stefan Hainzl (Co-author), Michael Ablinger (Co-author), Johann Bauer (Co-author), Julia Reichelt (Co-author), Ulrich Koller* (Last author)

*Corresponding author for this work

Research output: Contribution to journalOriginal Article (Journal)peer-review

62 Citations (Web of Science)
Original languageEnglish
Pages (from-to)2585-2598
JournalMOLECULAR THERAPY
Volume25
Issue number11
DOIs
Publication statusPublished - 2017

Keywords

  • EPIDERMOLYSIS-BULLOSA SIMPLEX
  • EPIDERMAL STEM-CELLS
  • GENE-THERAPY
  • KERATINOCYTES
  • TRANSPLANTATION
  • ENDONUCLEASE
  • KERATIN-14
  • DISORDERS
  • FAMILY
  • SAFETY

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