Prospects for human gene therapy

Abstract

Procedures have now been developed for inserting functional genes into the bone marrow of mice. The most effective delivery system at present uses retroviral-based vectors to transfer a gene into murine bone marrow cells in culture. The genetically altered bone marrow is then implanted into recipient animals. These somatic cell gene therapy techniques are becoming increasingly efficient. Their future application in humans should result in at least partial correction of a number of genetic disorders. However, the safety of the procedures must still be established by further animal studies before human clinical trials would be ethical.

KIE: Experimental gene therapy has been carried out in fruit flies and mice, and future clinical trials in humans are being debated among scientists and policy makers. Anderson, chief of the Laboratory of Molecular Hematology at the National Heart, Lung, and Blood Institute, examines how soon gene therapy might be available for the treatment of diseases in humans, and what criteria should be met before experimentation with human subjects begins. He identifies delivery, gene expression, and safety as the three requisites that must be satisfied, and reviews the animal work that has been done in these areas. Human trials should begin only after more animal studies have demonstrated to local and federal review committees that gene therapy is safe and offers the possibility of benefit to patients.