Is Gene Therapy a New Form of Medicine?
Despite semantics and anecdotal historical precedents, there was a general consensus among the participants that gene therapy is a novel form of medicine. The concept of gene therapy is simple: introduce genes whose products either can correct a defect, and thereby ameliorate the disease, or can slow the progression of a disease. Some researchers trivialize gene therapy as a mere tool for protein delivery; as such, it is merely a new delivery technology, akin to a pump. Whatever gene therapy may turn out to be, it is generally believed that its success will depend largely on the efficacy with which genes can be delivered to the desired cell, tissue, or organ. Nick Lemoine suggested that the term therapy is better than transfer, because this ensures regulatory and ethical consideration of clinical benefit rather than just safety. He also suggested that gene therapy will let us aim at targets against which it may take a long time to develop small-molecule drugs. Therefore the direct intr
