Designing Ethical Trials of Germline Gene Editing

Published In

The New England Journal of Medicine

Document Type


Publication Date



The recent announcement that researchers have successfully used the CRISPR (clustered regularly interspaced short palindromic repeats) gene-editing technique to correct a mutation that leads to hypertrophic cardiomyopathy in human embryos is the latest reminder of the urgency of the social and ethical issues surrounding potential clinical use of gene editing. Clinical use in humans is still far off; much research is needed to determine whether germline gene editing can be done safely and with acceptable risks; the legal and regulatory status of the technology varies widely around the world. Nevertheless, the drastic reduction in rates of off-target effects and mosaicism in the recent study — only 2 years since the first such experiment on human embryos was announced — shows how quickly the technology is progressing.



Persistent Identifier