Bioethics

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Genome editing, a technique by which DNA is inserted, replaced, or removed at particular locations in the human genome in order to correct mutations in the genomes that cause cancer, rare inherited disorders, or other diseases, is a promising new technology that may provide important, perhaps life-saving, treatment for patients.

Genome editing of somatic, non-germline cells, leads to modifications that are not passed to down subsequent generations, whereas genome editing of the germline results in heritable changes.

Our Position

ARM fully supports the therapeutic use of genome editing technologies of somatic cells, however, germline genome-editing techniques have not advanced to the point where human trials would be appropriate. As the science evolves, numerous criteria regarding the safety, legality, and ethics of germline genome editing must be met before human trials could begin.

ARM believes scientific research experiments on germline genome editing that stop short of implantation are needed to further understand many of the important safety issues associated with germline genome editing. We also strongly support and encourage the continued efforts to engage appropriate stakeholder groups from around the world—including patients, scientists, bioethicists, regulatory authorities, and various international governing bodies—in a public, science-based dialogue about the ethical and legal implications of genome editing.

Scientifically-grounded education on this topic is of utmost importance to broaden awareness of the immensely positive therapeutic potential of genome editing in somatic, non-heritable cells, and to distinguish those efforts from germline genome editing, and associated possible non-therapeutic approaches.

Background

Genome editing technologies (including homing endonucleases, AAV, zinc finger nucleases, CRISPR/Cas9 and TALENs) represent a powerful new approach for targeting and changing DNA sequences.

Genome editing has been successfully applied to the improvement of plants, the generation of transgenic animal models, to limit the propagation of harmful species (i.e.: malaria-transmitting mosquitos), and the development of potential therapeutics for human patients. Across a variety of academic and commercial settings, numerous efforts are currently underway to apply genome editing approaches to treat and potentially cure many human diseases, including HIV/AIDS, hemophilia, sickle cell disease, and several forms of cancer.

These efforts focus on the genomic repair of somatic (non-reproductive) cells and do not involve the genetic modification or manipulation of human embryos or reproductive cells (germline cells).

Somatic Versus
Germline Editing

Somatic cells account for the majority of cells in the human body—our internal organs, skin, bones, blood and connective tissue are all made up of somatic cells. Most genetic diseases manifest and can be treated in somatic cells. The DNA in these cells is non-heritable, which means that the editing modifications would affect only the patient and not be transmitted to his or her progeny.

Germline cells are those involved in reproduction (i.e. sperm or egg cells); because of this, genome editing approaches that target germline cells permanently change the DNA, the hereditary information that is passed along to future generations. Given that many important safety, ethical, and legal issues involved with this type of genome editing remain unresolved, ARM does not support clinical trials of human germline modifications at this time.

ARM also believes that at this time, given the current scientific knowledge and social environment surrounding gene editing, patients will benefit more immediately from resources being directed towards somatic genome editing technologies.

ARM’s member companies and organizations remain solely focused on somatic cell approaches to treatments and cures.

Additional Resources