A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaperJournal articleResearchpeer-review

  • Christopher A Vakulskas, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Daniel P Dever, Department of Pediatrics, Stanford University, Stanford, California, USA.
  • ,
  • Garrett R Rettig, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Rolf Turk, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Ashley M Jacobi, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Michael A Collingwood, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Nicole M Bode, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Matthew S McNeill, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Shuqi Yan, Integrated DNA Technologies, Inc., Coralville, IA, USA.
  • ,
  • Joab Camarena, Department of Pediatrics, Stanford University, Stanford, California, USA.
  • ,
  • Ciaran M Lee, Department of Bioengineering, Rice University, Houston, TX, USA.
  • ,
  • So Hyun Park, Department of Bioengineering, Rice University, Houston, TX, USA.
  • ,
  • Volker Wiebking, Department of Pediatrics, Stanford University, Stanford, California, USA.
  • ,
  • Rasmus O Bak
  • Natalia Gomez-Ospina, Department of Pediatrics, Stanford University, Stanford, California, USA.
  • ,
  • Mara Pavel-Dinu, Department of Pediatrics, Stanford University, Stanford, California, USA.
  • ,
  • Wenchao Sun, Biomaterials and Advanced Drug Delivery Laboratory, Stanford University School of Medicine, Palo Alto, CA, USA.
  • ,
  • Gang Bao, Department of Bioengineering, Rice University, Houston, TX, USA.
  • ,
  • Matthew H Porteus, Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. Electronic address: mporteus@stanford.edu.
  • ,
  • Mark A Behlke, Integrated DNA Technologies, Inc., Coralville, IA, USA. mbehlke@idtdna.com.
Original languageEnglish
JournalNature Medicine
Volume24
Issue8
Pages (from-to)1216-1224
Number of pages9
ISSN1078-8956
DOIs
Publication statusPublished - 2018

    Research areas

  • BONE-MARROW, CRISPR-CAS9 NUCLEASES, GENOME, GUIDE RNA, HUMAN T-CELLS, IN-VIVO, MAMMALIAN-CELLS, MARROW TRANSPLANTATION, MESSENGER-RNA, OFF-TARGET

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ID: 130916262