Human Gene Editing Research: Is the Future Here Yet?

Abstract

Since the discovery of DNA, researchers have pursued the prospect of correcting genetic disorders using genetic interventions. The most recent development, gene editing, poses many scientific, medical, ethical, and policy challenges, especially when the goal is editing the genomes of embryos, creating changes that can be inherited by future generations. Genetic treatments for already-born persons are not controversial, but inheritable genetic changes raise concerns about dangerous outcomes, questions about how to prioritize among scientific and societal needs, and worries about pursuing genetic changes that are enhancements rather than treatments for disease. The history of genetic-intervention research and the development of gene-editing tools like CRISPR were complicated enough, even before the “CRISPR babies” controversy arose in late 2018. CRISPR and related editing technologies should be used for basic research in order to learn more about human development and disease, but there is considerable disagreement and reason to be cautious about clinical applications. Moreover, no global enforcement mechanism exists to detect and prevent deviations from policy. Improved transparency, robust ongoing discussion, and increased education in ethics and genetics for scientists, students, and the public may therefore be both achievable goals and best practices for this rapidly developing science.