Human Genome Editing: An Evolving Regulatory Climate

Abstract

Human genome editing is the most consequential genetic technology for precision medicine to emerge in many years. Although genome-editing techniques have been available for more than a decade, the recently developed CRISPR-Cas9 system, in particular, has become the genome-editing technology of choice because of its ease of use and efficiency. Theoretically, the uses of genome-editing technology in humans can be divided into two major branches: the use in somatic cells for treating or preventing disease, and the use in gametes or embryos for research or germline modification in human reproduction. From that division, these branches diverge in terms of ethical, social and political impacts, and they also diverge with respect to regulatory responses from governments and professional organizations and the development of consensus norms. Already, genome-editing technologies are in human clinical trials, and genome-editing experiments on viable human embryos have been conducted. This article will situate genome editing in the lineage of human genetic modification technologies that originated in the 1970s. It will then briefly describe the major branches of application for human genome-editing technology, summarize the state of technical developments as of this writing, and provide a short overview of the relevant U.S. regulatory responses to date and those that may develop in the future.

Keywords: Genome Editing, Gene Editing, CRISPR, CRISPR-Cas9, DNA, Gene Therapy, Genetics, Germline Modification, RAC, FDA