Abstract
Vol.36 No.2
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Human germline genome editing therapy
JMOR, 36(2) 99-105, 2019
Genome editing is an ideal strategy for gene therapy to treat various disorders. In contrast to the overexpression of therapeutic cDNA, genome editing enables precise gene repair as well as ensures the stable and regulated expression of the edited gene. The recent development of artificial nucleases, such as CRISPR-Cas9, has made it possible to overcome the low efficiency of targeted DNA integration, gene repair and gene knockout, which are not feasible with conventional gene addition therapy. Genome editing has already been used in clinical applications for cancer and AIDS therapy, where the benefits outweigh the associated risks. However, genome editing using donor DNA for homology-directed repair (HDR) still has challenges with regard to its efficiency and safety. Off-target mutations have not been fully evaluated concerning the infidelity of nucleases or random chromosomal integration of donor DNA. In contrast to somatic genome editing, which can be regulated similarly to conventional gene therapy, heritable genome editing must be considered more carefully from therapeutic as well as ethical aspects. The recent birth of genome-edited twin girls in China shocked the world and has ignited international debate on how to regulate this exciting but potentially dangerous technology.
