Ethics

Research in Gene Therapy & Genetic Engineering

Code of Medical Ethics Opinion 7.3.6

Gene therapy involves the replacement or modification of a genetic variant to restore or enhance cellular function or the improve response to nongenetic therapies. Genetic engineering involves the use of recombinant DNA techniques to introduce new characteristics or traits. In medicine, the goal of gene therapy and genetic engineering is to alleviate human suffering and disease. As with all therapies, this goal should be pursued only within the ethical traditions of the profession, which gives primacy to the welfare of the patient.

In general, genetic manipulation should be reserved for therapeutic purposes. Efforts to enhance “desirable” characteristics or to “improve” complex human traits are contrary to the ethical tradition of medicine. Because of the potential for abuse, genetic manipulation of nondisease traits or the eugenic development of offspring may never be justifiable.

Moreover, genetic manipulation can carry risks to both the individuals into whom modified genetic material is introduced and to future generations. Somatic cell gene therapy targets nongerm cells and thus does not carry risk to future generations. Germ-line therapy, in which a genetic modification is introduced into the genome of human gametes or their precursors, is intended to result in the expression of the modified gene in the recipient’s offspring and subsequent generations. Germ-line therapy thus may be associated with increased risk and the possibility of unpredictable and irreversible results that adversely affect the welfare of subsequent generations.

Thus in addition to fundamental ethical requirements for the appropriate conduct of research with human participants, research in gene therapy or genetic engineering must put in place additional safeguards to vigorously protect the safety and well-being of participants and future generations.

Physicians should not engage in research involving gene therapy or genetic engineering with human participants unless the following conditions are met:

(a) Experience with animal studies is sufficient to assure that the experimental intervention will be safe and effective and its results predictable.

(b) No other suitable, effective therapies are available.

(c) Gene therapy is restricted to somatic cell interventions, in light of the far-reaching implications of germ-line interventions.

(d) Evaluation of the effectiveness of the intervention includes determination of the natural history of the disease or condition under study and follow-up examination of the participants’ descendants.

(e) The research minimizes risks to participants, including those from any viral vectors used.

(f) Special attention is paid to the informed consent process to ensure that the prospective participant (or legally authorized representative) is fully informed about the distinctive risks of the research, including use of viral vectors to deliver the modified genetic material, possible implications for the participant’s descendants, and the need for follow-up assessments.

Physicians should be aware that gene therapy or genetic engineering interventions may require additional scientific and ethical review, and regulatory oversight, before they are introduced into clinical practice.

AMA Principles of Medical Ethics: I, V, VII

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