Gene Therapy for Cystic Fibrosis

Abstract: A 5 page paper discussing the status of gene therapy for the treatment or prevention of cystic fibrosis. Cystic fibrosis is an autosomal recessive disorder caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator protein (CFTR). Mutations at this site lead to a variety of associated symptoms, and those with the disease have an expected life span of only 25 years. Gene therapy has had successful clinical trials in adults, but the real debate centers around the possibility (and ethics) of in utero gene therapy. Bibliography lists 9 sources.

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Catagory: Disease, Treatment, & Epidemiology

Subcatagory: Medicine, Medical Issues, & Pharmacy

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