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

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    Author
    Nicol, D; Eckstein, L; Morrison, M; Sherkow, JS; Otlowski, M; Whitton, T; Bubela, T; Burdon, KP; Chalmers, D; Chan, S; ...
    Date
    2017-09-25
    Source Title
    Genome Medicine: medicine in the post-genomic era
    Publisher
    BIOMED CENTRAL LTD
    University of Melbourne Author/s
    Mackey, David
    Affiliation
    Ophthalmology (Eye & Ear Hospital)
    Metadata
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    Document Type
    Journal Article
    Citations
    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 Access
    URI
    http://hdl.handle.net/11343/255116
    DOI
    10.1186/s13073-017-0475-4
    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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