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

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Nicol, D; Eckstein, L; Morrison, M; Sherkow, JS; Otlowski, M; Whitton, T; Bubela, T; Burdon, KP; Chalmers, D; Chan, S; ...Date
2017-09-25Source Title
Genome Medicine: medicine in the post-genomic eraPublisher
BIOMED CENTRAL LTDUniversity of Melbourne Author/s
Mackey, DavidAffiliation
Ophthalmology (Eye & Ear Hospital)Metadata
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Journal ArticleCitations
Nicol, 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 Status
Open AccessAbstract
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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