Article
Cystic Fibrosis Gene Therapy in the UK and Elsewhere.
Human gene therapy - 1 May 2015
Griesenbach Uta, Pytel Kamila M, Alton Eric W F W
Abstract excerpt
The cystic fibrosis transmembrane conductance regulator (CFTR) gene was identified in 1989. This opened the door for the development of cystic fibrosis (CF) gene therapy, which has been actively pursued for the last 20 years. Although 26 clinical trials involving approximately 450 patients have been carried out, the vast majority of these trials were short and included small numbers of patients; they were not...
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