Key Challenges in Bringing CRISPR-Mediated Somatic Cell Therapy into the Clinic
Author(s)
Nicol, Dianne
Eckstein, Lisa
Morrison, Michael
Sherkow, Jacob S.
Otlowski, Margaret
Whitton, Tess
Bubela, Tania
Burdon, Kathryn P.
Chalmers, Don
Chan, Sarah
Charlesworth, Jac
Critchley, Christine
Crossley, Merlin
de Lacey, Sheryl
Dickinson, Joanne L.
Hewitt, Alex W.
Kamens, Joanne
Kato, Kazuto
Kleiderman, Erika
Kodama, Satoshi
Liddicoat, John
Mackey, David A.
Newson, Ainsley J.
Nielsen, Jane
Wagner, Jennifer K.
McWhirter, Rebekah E.
Issue Date
2017-09-25
Keyword(s)
Somatic Cell Therapy
Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR)
Genome Editing
Stakeholder Engagement Mechanisms
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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