Somatic Genome Editing for Health: Disease Treatments and Beyond

Abstract

Genome editing has facilitated versatile gene modifications in somatic and stem cells. Despite the early stage, therapeutic uses of ZFN and TALEN have already demonstrated promising results in the treatment of HIV and leukemia. Although the medical conditions to which genome editing therapy is applied are being expanded in the clinical trials, it is currently unclear whether regulatory authorities will approve genome editing therapy in the near future. Moreover, the widespread use of CRISPR/ Cas9, in particular, is likely to lead to clinically unproven disease treatments. Furthermore, genome editing might be used for gene doping or cosmetic treatments. This article discusses the prospects of somatic genome editing for health purposes, while briefly reviewing the relevant clinical trials. Moreover, some potential ethical issues arising from the widespread use of genome editing are considered to discuss how genome-editing medicine might be appropriately integrated into the global society.