title: Progress and prospects: gene therapy clinical trials (part 1).
abstract: 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.
authors.forename: B L
authors.initials: BL
authors.lastname: Alexander
authors.forename: R R
authors.initials: RR
authors.lastname: Ali
authors.forename: E W F
authors.initials: EW
authors.lastname: Alton
authors.forename: J W
authors.initials: JW
authors.lastname: Bainbridge
authors.forename: S
authors.initials: S
authors.lastname: Braun
authors.forename: S H
authors.initials: SH
authors.lastname: Cheng
authors.forename: T R
authors.initials: TR
authors.lastname: Flotte
authors.forename: H B
authors.initials: HB
authors.lastname: Gaspar
authors.forename: M
authors.initials: M
authors.lastname: Grez
authors.forename: U
authors.initials: U
authors.lastname: Griesenbach
authors.forename: M G
authors.initials: MG
authors.lastname: Kaplitt
authors.forename: M G
authors.initials: MG
authors.lastname: Ott
authors.forename: R
authors.initials: R
authors.lastname: Seger
authors.forename: M
authors.initials: M
authors.lastname: Simons
authors.forename: A J
authors.initials: AJ
authors.lastname: Thrasher
authors.forename: A Z
authors.initials: AZ
authors.lastname: Thrasher
authors.forename: S
authors.initials: S
authors.lastname: Ylä-Herttuala
mesh_terms: D002986:Clinical Trials as Topic; D003327:Coronary Disease / Q000628:therapy; D003550:Cystic Fibrosis / Q000628:therapy; D005128:Eye Diseases / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D006105:Granulomatous Disease, Chronic / Q000628:therapy; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy; D010300:Parkinson Disease / Q000628:therapy; D016491:Peripheral Vascular Diseases / Q000628:therapy; D016511:Severe Combined Immunodeficiency / Q000628:therapy; D019896:alpha 1-Antitrypsin Deficiency / Q000628:therapy