Key challenges in bringing CRISPR-ediated somatic cell therapy into the clinic
AuthorNicol, D; Eckstein, L; Morrison, M; Sherkow, JS; Otlowski, M; Whitton, T; Bubela, T; Burdon, KP; Chalmers, D; Chan, S; ...
Source TitleGenome Medicine: medicine in the post-genomic era
PublisherBIOMED CENTRAL LTD
University of Melbourne Author/sMackey, David
AffiliationOphthalmology (Eye & Ear Hospital)
Document TypeJournal Article
CitationsNicol, D., Eckstein, L., Morrison, M., Sherkow, J. S., Otlowski, M., Whitton, T., Bubela, T., Burdon, K. P., Chalmers, D., Chan, S., Charlesworth, J., Critchley, C., Crossley, M., de Lacey, S., Dickinson, J. L., Hewitt, A. W., Kamens, J., Kato, K., Kleiderman, E. ,... McWhirter, R. E. (2017). Key challenges in bringing CRISPR-ediated somatic cell therapy into the clinic. GENOME MEDICINE, 9 (1), https://doi.org/10.1186/s13073-017-0475-4.
Access StatusOpen Access
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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