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Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic
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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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Official URL: http://dx.doi.org/10.1186/s13073-017-0475-4
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.
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