Genetic therapy. Past, present, and future.

摘要

The early stages of genetic therapy present challenges for clinicians and basic scientists. Clinicians must become familiar with new terminology and concepts, and must keep a perspective on the new field in the face of inflated claims and high-profile failures. Basic scientists must continually return to disease models and to patients to determine what are the proper safety issues and relevant efficacy questions for specific diseases and vector systems. And in an era of instant information, all concerned parties must be careful about how progress is communicated to colleagues, patients, and the lay public.