Patients Suffer While the Science Establishment Resists Innovative Therapies

Abstract

Human gene therapy has been up to now of a type that affects only the patient being treated; it has not modified sperm or eggs cells or embryos in a way that would constitute “germ line gene therapy” (GLGT) by creating a heritable change and affecting future generations.  Preclinical research has progressed almost to the point where GLGT interventions will be possible with a reasonable likelihood of success, but such clinical trials are currently prohibited: NIH's Recombinant DNA Advisory Committee is not permitted even to consider such proposals, and the FDA cannot use appropriated funds to review such trials.  Such absolute prohibitions are bad for patients and bad public policy.