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


Nicol, D and Eckstein, L and Morrison, M and Sherkow, JS and Otlowski, M and Whitton, T and Bubela, T and Burdon, KP and Chalmers, D and Chan, S and Charlesworth, J and Critchley, C and Crossley, M and de Lacey, S and Dickinson, JL and Hewitt, AW and Kamens, J and Kato, K and Kleiderman, E and Kodama, S and Liddicoat, J and Mackey, DA and Newson, AJ and Nielsen, J and Wagner, JK and McWhirter, RE, Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic, Genome Medicine, 9, (1) Article 85. ISSN 1756-994X (2017) [Contribution to Refereed Journal]


Copyright Statement

Copyright 2017 The Authors. Licensed under Creative Commons Attribution 4.0 International (CC BY 4.0)

DOI: doi:10.1186/s13073-017-0475-4


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 CRISPRmediated somatic cell therapy.

Item Details

Item Type:Contribution to Refereed Journal
Keywords:genetics, ethics
Research Division:Medical and Health Sciences
Research Group:Clinical Sciences
Research Field:Medical Genetics (excl. Cancer Genetics)
Objective Division:Health
Objective Group:Clinical Health (Organs, Diseases and Abnormal Conditions)
Objective Field:Cancer and Related Disorders
Author:Nicol, D (Professor Dianne Nicol)
Author:Eckstein, L (Dr Lisa Eckstein)
Author:Otlowski, M (Professor Margaret Otlowski)
Author:Whitton, T (Mr Thomas Whitton)
Author:Burdon, KP (Associate Professor Kathryn Burdon)
Author:Chalmers, D (Professor Don Chalmers)
Author:Charlesworth, J (Dr Jac Charlesworth)
Author:Dickinson, JL (Associate Professor Joanne Dickinson)
Author:Hewitt, AW (Professor Alex Hewitt)
Author:Mackey, DA (Professor David Mackey)
Author:Nielsen, J (Dr Jane Nielsen)
Author:McWhirter, RE (Dr Rebekah McWhirter)
ID Code:121348
Year Published:2017
Web of Science® Times Cited:1
Deposited By:Menzies Institute for Medical Research
Deposited On:2017-09-26
Last Modified:2018-12-13
Downloads:44 View Download Statistics

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