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

UMMS Affiliation

Gene Therapy Center; Department of Pediatrics



Document Type


Medical Subject Headings

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


Allergy and Immunology | Genetics and Genomics | Pediatrics | Respiratory Tract Diseases


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.

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Citation: Gene Ther. 2007 Oct;14(20):1439-47. Link to article on publisher's site

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Link to Article in PubMed

PubMed ID