Abstract
Editorial summaryGenome 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.
Highlights
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
We identify some of the major challenges for clinicians, regulators, and human research ethics committees in the clinical translation of CRISPRmediated somatic cell therapy
Regulatory challenges for CRISPR-mediated somatic cell therapy The discovery that clustered regularly interspersed short palindromic repeats (CRISPR) and CRISPR-associated proteins found naturally in prokaryotic cells can be used to alter the genome of living organisms—including humans—has been one of the most exciting breakthroughs in biomedical science
Summary
Key challenges in bringing CRISPRmediated somatic cell therapy into the clinic Dianne Nicol1*, Lisa Eckstein, Michael Morrison, Jacob S.
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