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Gene therapy for cystic fibrosis
Author(s) -
Davies Jane C.,
Geddes Duncan M.,
Alton Eric W. F. W.
Publication year - 2001
Publication title -
the journal of gene medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.689
H-Index - 91
eISSN - 1521-2254
pISSN - 1099-498X
DOI - 10.1002/jgm.200
Subject(s) - cystic fibrosis , cystic fibrosis transmembrane conductance regulator , genetic enhancement , gene transfer , regulator , medicine , clinical trial , gene , bioinformatics , computational biology , biology , genetics
Abstract Cystic fibrosis (CF) is associated with significant morbidity and mortality, despite significant advances in conventional treatment. The field of gene therapy has progressed rapidly since the cystic fibrosis transmembrane conductance regulator (CFTR) gene was cloned. In this review we discuss current knowledge on the underlying molecular defect in CF, and the progress in gene transfer studies from the early in vitro work through to clinical trials, including the development of endpoints to assess efficacy. We highlight the problems encountered, and likely future directions of the field. Copyright © 2001 John Wiley & Sons, Ltd.