Status of gene therapy for cystic fibrosis lung disease

Abstract excerpt

Gene therapy for the treatment of cystic fibrosis should be a “natural”: Cystic fibrosis (CF) is a recessive disease associated with loss of function mutations in the CF transmembrane conductance regulator (CFTR) gene, which has a well-characterized gene product; heterozygotes, as predicted, appear to be phenotypically perfectly normal; the level of expression of CFTR in affected cells generally appears to be...

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