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

Nicol, D ORCID: 0000-0002-6553-2839, Eckstein, L ORCID: 0000-0002-7161-7521, Morrison, M, Sherkow, JS, Otlowski, M ORCID: 0000-0003-4707-4325, Whitton, T, Bubela, T, Burdon, KP ORCID: 0000-0001-8217-1249, Chalmers, D ORCID: 0000-0002-7925-8818, Chan, S, Charlesworth, J ORCID: 0000-0001-6201-3518, Critchley, C, Crossley, M, de Lacey, S, Dickinson, JL ORCID: 0000-0003-4621-1703, Hewitt, AW ORCID: 0000-0002-5123-5999, Kamens, J, Kato, K, Kleiderman, E, Kodama, S, Liddicoat, J, Mackey, DA, Newson, AJ, Nielsen, J ORCID: 0000-0002-3005-0047, Wagner, JK and McWhirter, RE ORCID: 0000-0002-9409-8074 2017 , 'Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic' , Genome Medicine, vol. 9, no. 1 , pp. 1-4 , doi: https://doi.org/10.1186/s13073-017-0475-4.

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Abstract

Genome editing using clustered regularly interspersedshort palindromic repeats (CRISPR) and CRISPR-associatedproteins offers the potential to facilitate safe and effectivetreatment of genetic diseases refractory to other typesof intervention. Here, we identify some of the majorchallenges for clinicians, regulators, and human researchethics committees in the clinical translation of CRISPRmediatedsomatic cell therapy.

Item Type: Article
Authors/Creators: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
Keywords: genetics, ethics
Journal or Publication Title: Genome Medicine
Publisher: BioMed Central Ltd.
ISSN: 1756-994X
DOI / ID Number: https://doi.org/10.1186/s13073-017-0475-4
Copyright Information:

Copyright 2017 The Authors. Licensed under Creative Commons Attribution 4.0 International (CC BY 4.0) https://creativecommons.org/licenses/by/4.0/

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