Human Genome Editing Science, Ethics, and Governance (2017) / Chapter Skim
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With these advances has come an explosion of interest in the possible applications of genome editing, both in conducting fundamental research and potentially in promoting human health through the treatment or prevention of disease and disability. The latter possibilities range from restoring normal function in diseased organs by editing somatic cells to preventing genetic diseases in future children and their descendants by editing the human germline.
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While genome editing has potential applications in agriculture and nonhuman animals, this committee's task was focused on human applications. The charge to the committee included elements pertaining to the state of the science in genome editing, possible clinical applications of these technologies, potential risks and benefits, whether standards can be established for quantifying unintended effects, whether current regulatory frameworks provide adequate oversight, and what overarching principles should guide the regulation of genome editing in humans.
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Clinical applications of genome editing that target somatic cells affect only the patient, and are akin to existing efforts to use gene therapy for disease treatment and prevention; they do not affect offspring. By contrast, germline interventions would be aimed at altering a genome in a way that would affect not only the resulting child but potentially some of the child's descendants as well.
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cells; and in the future might be usable for clinical applications involving reproductive cells, which would produce heritable changes. Basic Science Laboratory Research Basic laboratory research involving genome editing of human cells and tissues is critical to advancing biomedical science.
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The idea of making genetic changes to somatic cells -- referred to as "gene therapy" -- is not new, and genome editing for somatic applications would be similar. Gene therapy has been governed by ethical norms and subject to regulatory oversight for some time, and this experience offers guidance for establishing similar norms and oversight mechanisms for genome editing of somatic cells.
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Nonetheless, the technology is of interest because thousands of inherited diseases are caused by mutations in single genes.3 Thus, editing the germline cells of individuals who carry these mutations could allow them to have genetically related children without the risk of passing on these conditions. Germline genome editing is unlikely to be used often enough in the foreseeable future to have a significant effect on the prevalence of these diseases but could provide some families with their best or most acceptable option for averting disease transmission, either because existing technologies, such as prenatal or preimplantation genetic diagnosis, will not work in some cases or because the existing technologies involve discarding affected embryos or using selective abortion following prenatal diagnosis.
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Policy in this area will require a careful balancing of cultural norms, the physical and emotional well-being of children, parental autonomy, and the ability of regulatory systems to prevent inappropriate or abusive applications. In light of the technical and social concerns involved, the committee concluded that heritable genome-editing research trials might be permitted, but only following much more research aimed at meeting existing risk/benefit standards for authorizing clinical trials and even then, only for compelling reasons and under strict oversight.
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Likewise, physicians and patients will interpret them in light of the specifics of individual cases for which germline genome editing may be considered as a possible option. Starting points for defining some of these concepts exist, such as the definition of "serious disease or condition" used by the FDA.5 Finally, those opposed to heritable editing may even conclude that, properly implemented, the above criteria are so strict that they would have the effect of preventing all clinical trials involving germline genome editing.
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As noted above, for somatic genome editing, it is essential that transparent and inclusive public policy debates precede any consideration of whether to authorize clinical trials for indications that go beyond treatment or prevention of disease or disability (e.g., for enhancement)
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Discussion is fostered by the various state and federal bioethics commissions, which typically bring together technical experts and social scientists in meetings that are open to the public. And the National Institutes of Health's Recombinant DNA Advisory Committee offers a venue for general public discussion of gene therapy, for review of specific protocols, and for transmission of advice to regulators.
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3. Due care: The principle of due care for patients enrolled in research studies or receiving clinical care requires proceeding carefully and deliberately, and only when supported by sufficient and robust evidence.
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equitable distribution of the burdens and benefits of research and (2) broad and equitable access to the benefits of resulting clinical applications of human genome editing.
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Ongoing reassessment and public participation should precede any clinical trials of heritable germline editing (7.2) Incorporate public participation into the human genome editing policy process concerning "enhancement" (7.3)
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