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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.


Article authors:

Nicol, Dianne and Eckstein, Lisa and Morrison, Michael and Sherkow, Jacob S. and Otlowski, Margaret and Whitton, Tess and Bubela, Tania and Burdon, Kathryn and Chalmers, Donald and Chan, Sarah and Charlesworth, Jacqueline and Critchley, Christine and Crossley, Merlin and de Lacey, Sheryl and Dickinson, Joanne and Hewitt, Alex and Liddicoat, Johnathon and Kamens, Joanne and Kato, Kazuto and Kleiderman, Erica and Kodama, Satoshi and Mackey, David and Newson, Ainsley and Nielsen, Jane L and Wagner, Jennifer and McWhirter, Rebekah, Key Challenges in Bringing CRISPR-Mediated Somatic Cell Therapy into the Clinic (September 25, 2017). Genome Medicine, 2017.