Governing, Protecting, and Regulating the Future of Genome Editing

Abstract

With the recent breakthroughs in genomics and advances in genome-editing techniques, most notably the discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9),1 the matter of genome editing and health innovation has become of particular importance in society. New genome-editing techniques hold considerable potential to enhance personalized medicine and deliver cures to conditions and diseases that currently cannot be tackled. However, considerable work remains to be done in order to realize this potential.Means to modify the human genome have been of interest to scientists for a considerable time and significant milestones were achieved during the second half of the last century.2 Several tools have existed prior to the discovery of Cas9, such as meganucleases, zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs).3 Already in 2003, the first commercial gene-editing therapy -Gendicine -was registered in China.4 Almost a decade later, in 2012, the first gene therapy medicinal product -Glybera