Human Genome Editing in the Clinic: New Challenges in Regulatory Benefit-Risk Assessment

Abstract

As genome editing rapidly progresses toward the realization of its clinical promise, assessing the suitability of current tools and processes used for its benefit-risk assessment is critical. Although current regulations may initially provide an adequate regulatory framework, improvements are recommended to overcome several existing technology-based safety and efficacy issues.

Keywords: benefit-risk assessment; clinical trials; efficacy; genome editing; off-target effects; regulations; safety.