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Review
. 2007 Oct;14(20):1439-47.
doi: 10.1038/sj.gt.3303001.

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

B L Alexander, R R Ali, E W F Alton, J W Bainbridge, S Braun, S H Cheng, T R Flotte, H B Gaspar, M Grez, U Griesenbach, M G Kaplitt, M G Ott, R Seger, M Simons, A J Thrasher, A Z Thrasher, S Ylä-Herttuala
Review

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

B L Alexander et al. Gene Ther. 2007 Oct.

Erratum in

  • Gene Ther. 2007 Dec;14(24):1754. multiple author names added

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.

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