Article
A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease.
Human gene therapy - 10 Oct 2001
Aitken M L, Moss R B, Waltz D A, Dovey M E, Tonelli M R, McNamara S C, Gibson R L, Ramsey B W, Carter B J, Reynolds T C
Abstract excerpt
Cystic fibrosis (CF) is one of the most common autosomal recessive disorders in North America, leading to significant morbidity and early mortality. The defect in the cystic fibrosis transmembrane conductance regulator protein (CFTR) function can be corrected in vitro by gene replacement with a wild-type gene. A Phase I, single administration, dose escalation trial was designed and executed to assess safety and...
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