Progress and prospects: gene therapy clinical trials (part 1)

Gene Ther. 2007 Oct;14(20):1439-47. doi: 10.1038/


Over the last two decades gene therapy has moved from preclinical to clinical studies for many diseases ranging from single gene disorders such as cystic fibrosis and Duchenne muscular dystrophy, to more complex diseases such as cancer and cardiovascular disorders. Gene therapy for severe combined immunodeficiency (SCID) is the most significant success story to date, but progress in many other areas has been significant. We asked 20 leaders in the field succinctly to summarize and comment on clinical gene therapy research in their respective areas of expertise and these are published in two parts in the Progress and Prospect series.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic*
  • Coronary Disease / therapy
  • Cystic Fibrosis / therapy
  • Eye Diseases / therapy
  • Genetic Therapy / methods
  • Genetic Therapy / trends*
  • Granulomatous Disease, Chronic / therapy
  • Humans
  • Lysosomal Storage Diseases / therapy
  • Muscular Dystrophy, Duchenne / therapy
  • Parkinson Disease / therapy
  • Peripheral Vascular Diseases / therapy
  • Severe Combined Immunodeficiency / therapy
  • alpha 1-Antitrypsin Deficiency / therapy