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 KodamaJohn Liddicoat, David A. Mackey, Ainsley J. Newson, Jane Nielsen, Jennifer K. Wagner, Rebekah E. McWhirter

Research output: Contribution to journalComment/debate

Abstract

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.

Original languageEnglish (US)
Article number85
JournalGenome medicine
Volume9
Issue number1
DOIs
StatePublished - Sep 25 2017
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Genetics(clinical)

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  • Cite this

    Nicol, D., Eckstein, L., Morrison, M., Sherkow, J. S., Otlowski, M., Whitton, T., Bubela, T., Burdon, K. P., Chalmers, D., Chan, S., Charlesworth, J., Critchley, C., Crossley, M., de Lacey, S., Dickinson, J. L., Hewitt, A. W., Kamens, J., Kato, K., Kleiderman, E., ... McWhirter, R. E. (2017). Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic. Genome medicine, 9(1), [85]. https://doi.org/10.1186/s13073-017-0475-4