Genome editing is not new. But new powerful, precise, and less costly genome editing tools, such as CRISPR/Cas9, have led to an explosion of new research opportunities and potential clinical applications, both heritable and
Human genome editing is already widely used in basic research and is in the early stages of development and trials for clinical applications that involve
However, there is significant public concern about the prospect of using these same techniques for
«Human genome editing holds tremendous promise for understanding, treating, or preventing many devastating genetic diseases, and for improving treatment of many other illnesses," said Alta Charo,
Germline genome editing, in contrast, is contentious because genetic changes would be inherited by the next generation. Many view germline editing as crossing an «ethically inviolable» line, the report says. Concerns raised include spiritual objections to interfering with human reproduction to speculation about effects on social attitudes toward people with disabilities to possible risks to the health and safety of future children. But germline genome editing could provide some parents who are carriers of genetic diseases with their best or most acceptable option for having genetically related children who are born free of these diseases.
Heritable germline editing is not ready to be tried in humans. Much more research is needed before it could meet the appropriate risk and benefit standards for clinical trials. The technology is advancing very rapidly, though, making heritable genome editing of early embryos, eggs, sperm, or precursor cells in the foreseeable future «a realistic possibility that deserves serious consideration," the report says. Although heritable germline genome editing trials must be approached with caution, the committee said, caution does not mean prohibition.
At present, heritable germline editing is not permissible in the United States, due to an ongoing prohibition on the
If current restrictions are removed, and for countries where germline editing would already be permitted, the committee recommended stringent criteria that would need to be met before going forward with clinical trials. They include: (1) absence of reasonable alternatives; (2) restriction to editing genes that have been convincingly demonstrated to cause or strongly predispose to a serious disease or condition; (3) credible
Policymaking surrounding human genome editing applications should incorporate public participation, and funding of genome editing research should include support to study the
The report recommends a set of overarching principles that should be used by any nation in governing human genome editing research or applications:
well-being— providing benefit and preventing harm to those affected
- Transparency — openness and sharing of information in ways that are accessible and understandable to patients, their families, and other stakeholders
- Due care — proceeding only when supported by sufficient and robust evidence
- Responsible science — adhering to the highest standards of research in accordance with international and professional norms
- Respect for persons — recognizing the personal dignity of all individuals and with respect for their decisions
- Fairness — treating all cases alike, with an equitable distribution of risks and benefits
- Transnational cooperation — committing to collaborative approaches for research and governance while respecting different cultural contexts.
«Genome editing research is very much an international endeavor, and all nations should ensure that any potential clinical applications reflect societal values and be subject to appropriate oversight and regulation," said committee
Such international coordination would enhance consistency of regulation.»
The study was funded by the Defense Advanced Research Projects Agency, the Greenwall Foundation, the John D. and Catherine T. MacArthur Foundation,
The National Academy of Sciences is a private, nonprofit institution that was established under a congressional charter signed by President Abraham Lincoln in 1863. It recognizes achievement in science by election to membership, and — with the National Academy of Engineering and the National Academy of Medicine — provides science, engineering, and health policy advice to the federal government and other organizations.Source: http://www.nasonline.org/news-and-multimedia/news/Human-Genome-Editing-Report.html