Publication Type Review
Authors Alexander B, Ali R, Alton E, Bainbridge J, Braun S, Cheng S, Flotte T, Gaspar H, Grez M, Griesenbach U, Kaplitt M, Ott M, Seger R, Simons M, Thrasher A, Thrasher A, Ylä-Herttuala S
Journal Gene Ther
Volume 14
Issue 20
Pagination 1439-47
Date Published 10/01/2007
ISSN 0969-7128
Keywords Clinical Trials as Topic, Genetic Therapy
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.
DOI 10.1038/sj.gt.3303001
PubMed ID 17909539
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