Gene Therapy: Promises, Problems and Prospects

Abstract

Gene therapy is a novel form of molecular medicine which will have a major impact on human health in the coming century. Although the advent of recombinant DNA technology in modern medicine will allow fetal genetic screening and genetic counseling, the vast majority of those born with the disease are likely to be helped by gene therapy approaches. The scope and definition of gene therapy have expanded in the past few years. In addition to the possibility of correcting inherited genetic disorders like cystic fibrosis, hemophilia and familial hypercholesterolemia, gene therapy approaches are being used to combat acquired diseases, like cancer, AIDS, infectious diseases, Parkinson’s disease, and Alzheimer’s disease. We are not, at this time, contemplating germ line gene therapy, due to the complex technical and ethical issues involved. We are interested in pursuing somatic cell gene therapy, which is exclusively for the benefit for the individual and cannot be passed on to the succeeding generation. The minimum requirement for gene therapy is sustained production of the therapeutic gene product without any harmful side effects (Anderson 1998; Verma and Somia 1997; Crystal 1995; Mulligan 1993; Leiden 1995).