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:10.1186/s13073-017-0475-4

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

Genome Med. 2017 Sep 25;9(1):85. doi: 10.1186/s13073-017-0475-4.

Authors

Dianne Nicol¹, Lisa Eckstein², Michael Morrison³, Jacob S Sherkow⁴, Margaret Otlowski², Tess Whitton^{2

5}, Tania Bubela⁶, Kathryn P Burdon⁵, Don Chalmers², Sarah Chan⁷, Jac Charlesworth⁵, Christine Critchley⁸, Merlin Crossley⁹, Sheryl de Lacey¹⁰, Joanne L Dickinson⁵, Alex W Hewitt⁵, Joanne Kamens¹¹, Kazuto Kato¹², Erika Kleiderman¹³, Satoshi Kodama¹⁴, John Liddicoat¹⁵, David A Mackey¹⁶, Ainsley J Newson¹⁷, Jane Nielsen², Jennifer K Wagner¹⁸, Rebekah E McWhirter^{2

5}

Affiliations

¹ Centre for Law and Genetics, Faculty of Law, University of Tasmania, Hobart, 7001, Australia. Dianne.Nicol@utas.edu.au.
² Centre for Law and Genetics, Faculty of Law, University of Tasmania, Hobart, 7001, Australia.
³ Centre for Health, Law and Emerging Technologies, Nuffield Department of Population Health, University of Oxford, Oxford, OX2 7DD, UK.
⁴ Innovation Center for Law and Technology, New York Law School, New York, 10013, USA.
⁵ Menzies Institute for Medical Research, University of Tasmania, Hobart, 7000, Australia.
⁶ Faculty of Health Sciences, Simon Fraser University, Burnaby, BC, V5A1S6, Canada.
⁷ Usher Institute for Population Health Sciences and Informatics, University of Edinburgh, Edinburgh, EH8 9YL, UK.
⁸ Department of Statistics, Data Science and Epidemiology, Swinburne University of Technology, Melbourne, 3122, Australia.
⁹ School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, 2052, Australia.
¹⁰ School of Nursing and Midwifery, Flinders University, Adelaide, 5042, Australia.
¹¹ Addgene, Cambridge, 02139, USA.
¹² Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Osaka, 565-0871, Japan.
¹³ Centre of Genomics and Policy, Department of Human Genetics, McGill University, Montreal, H3A0G4, Canada.
¹⁴ Department of Ethic, Graduate School of Letters, Kyoto University, Kyoto, 606-8501, Japan.
¹⁵ Faculty of Law, University of Cambridge, Cambridge, CB3 9DZ, UK.
¹⁶ Centre for Ophthalmology and Visual Science, University of Western Australia, Lions Eye Institute, Perth, 6009, Australia.
¹⁷ Sydney Health Ethics, Sydney School of Public Health, University of Sydney, Sydney, 2006, Australia.
¹⁸ Center for Translational Bioethics & Health Care Policy, Geisinger Health System, Danville, PA, 17822, USA.

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.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Biomedical Technology
Cell- and Tissue-Based Therapy* / economics
Cell- and Tissue-Based Therapy* / ethics
Clinical Medicine / economics
Clinical Medicine / legislation & jurisprudence
Clinical Medicine / trends
Clustered Regularly Interspaced Short Palindromic Repeats*
Humans
Intellectual Property