History of Gene Therapy

Abstract

Once a dream cure for genetic diseases, gene therapy came to be considered potentially realizable with the advent of recombinant DNA technology in the 1970s. In the 1980s, viral vectors were developed, and ethical issues regarding human gene manipulation began to be addressed. In 1990, the world’s first gene therapy was launched in the United States. Since then, various gene therapies have been tried around the world, but have failed to show clear efficacy. In addition, safety problems, including a fatal accident and the development of leukemia, have occurred with gene therapy. Consequently, the once high expectations for gene therapy declined. Since the end of the 2000s, however, the efficacy of gene therapies for treatment of genetic diseases and cancer has been confirmed time and again, and expectations for gene therapy have been revived. At present, more than 20 gene therapy products have been approved, and gene therapies are now poised to become practical medicine.