Genome Medicine (Sep 2017)

Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic

  • Dianne Nicol,
  • Lisa Eckstein,
  • Michael Morrison,
  • Jacob S. Sherkow,
  • Margaret Otlowski,
  • Tess Whitton,
  • Tania Bubela,
  • Kathryn P. Burdon,
  • Don Chalmers,
  • Sarah Chan,
  • Jac Charlesworth,
  • Christine Critchley,
  • Merlin Crossley,
  • Sheryl de Lacey,
  • Joanne L. Dickinson,
  • Alex W. Hewitt,
  • Joanne Kamens,
  • Kazuto Kato,
  • Erika Kleiderman,
  • Satoshi Kodama,
  • John Liddicoat,
  • David A. Mackey,
  • Ainsley J. Newson,
  • Jane Nielsen,
  • Jennifer K. Wagner,
  • Rebekah E. McWhirter

DOI
https://doi.org/10.1186/s13073-017-0475-4
Journal volume & issue
Vol. 9, no. 1
pp. 1 – 4

Abstract

Read online

Editorial summary Genome editing using clustered regularly interspersed short palindromic repeats (CRISPR) and CRISPR-associated proteins offers the potential to facilitate safe and effective treatment of genetic diseases refractory to other types of intervention. Here, we identify some of the major challenges for clinicians, regulators, and human research ethics committees in the clinical translation of CRISPR-mediated somatic cell therapy.