Scientific Publications by FDA Staff

IDrugs 2005 Dec;8(12):993-6

Challenges and future prospects in gene therapy.

Byrnes AP

Byrnes AP, US FDA, Div Cellular & Gene Therapies, Ctr Biol Evaluat & Res, Rockville, MD 20852 USA US FDA, Div Cellular & Gene Therapies, Ctr Biol Evaluat & Res, Rockville, MD 20852 USA

Gene therapy is a promising technique for treating disease through the modification of gene expression. It is currently being tested not only for correcting genetic defects, but also for treating cancer and other acquired diseases. Although this field is still relatively young, evidence for clinical efficacy has been observed and continued progress seems assured, as clinical trials continue to yield insights into how gene therapy can be applied and improvements are made in gene therapy tools.