Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic
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Nicol, Dianne
Eckstein, Lisa
Morrison, Michael
Sherkow, Jacob S.
Otlowski, Margaret
Whitton, Tess
Bubela, Tania
Burdon, Kathryn P.
Chalmers, Don
Chan, Sarah
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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.
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Genome Medicine
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