Clinical applications of germline genome editing are now possible and there is an urgent need to examine the potential of this new technology. Many scientific and medical questions about the procedures remain to be answered, and determining the safety and efficacy of germline genome editing will be necessary but not sufficient conditions for future clinical usage. There is a need for a framework to inform the development of a potential pathway from research to clinical use, recognizing that components of this framework may need to be periodically revised in response to our rapidly evolving knowledge. In addition, other important discussions are ongoing internationally about the implications for society of human germline genome editing and include issues such as access, equity, and consistency with religious views.
An international commission will be convened with the participation of National Academies of Sciences and Medicine throughout the world to develop a framework for considering technical, scientific, medical, regulatory, and ethical requirements for germline genome editing, should society conclude such applications are acceptable.
The U.S. National Academies of Sciences and Medicine and the UK Royal Society will serve as the commission’s secretariat.
Specifically, the commission will:
1) identify the scientific
issues (as well as societal and ethical issues, where inextricably linked to research and clinical practice) that
must be evaluated for various classes of possible applications. Potential applications considered
should range from genetic correction of severe, highly penetrant monogenic
diseases to various forms of genetic enhancement;
2) identify appropriate protocols and pre-clinical validation
for assessing and evaluating on-target and off-target events and any potential
developmental and long-term side effects;
3) identify appropriate protocols for assessing and evaluating
potential mosaicism and long-term implications;
4) identify ways to assess the balance between potential
benefits and harms to a child produced by genome editing and to subsequent
generations.
5) design appropriate protocols for obtaining consent from
patients, for obtaining ethical approval from knowledgeable review committees,
and for satisfying regulatory authorities;
6) identify and assess possible mechanisms for the long term
monitoring of children born with edited genomes; and
7) outline the research and clinical characteristics developed in tasks 1-6 that would form part of an oversight structure, including defining scientific criteria
for establishing where heritable genome editing might be appropriate,
overseeing any human clinical use, and bringing forward concerns about human
experiments.
