title: Cystic fibrosis: a look into the future of prenatal screening and therapy.
abstract: Despite recent guidelines suggesting prenatal screening for carriers of cystic fibrosis (CF) mutations, many physicians do not offer patients this service or even counseling. Some argue that the risks of miscarriage associated with prenatal diagnostic techniques outweigh the benefit of added insight, but with the advent of newer, noninvasive techniques, risks of miscarriage may be significantly lowered. Prenatal diagnosis provides parents the time to prepare for raising a child with CF, and soon, could provide treatment options in utero that could improve quality of life. Here, we describe two of the most promising gene therapy approaches: lentivirus and adenoassociated virus (AAV)-mediated gene transduction. Thus, prenatal detection and treatment is in a most crucial stage for care of patients with CF.
authors.affiliation: Georgetown University School of Medicine, Georgetown University Special Master's Program in Physiology, NW, Med/Dent NE, Washington, DC.
authors.forename: Kevin
authors.initials: K
authors.lastname: Nishida
authors.forename: Zachary
authors.initials: Z
authors.lastname: Smith
authors.forename: Dane
authors.initials: D
authors.lastname: Rana
authors.forename: Jereme
authors.initials: J
authors.lastname: Palmer
authors.forename: G Ian
authors.initials: GI
authors.lastname: Gallicano
mesh_terms: D003550:Cystic Fibrosis / Q000235:genetics* / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D000229:Dependovirus; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics; D006801:Humans; D016086:Lentivirus; D011296:Prenatal Diagnosis / Q000009:adverse effects / Q000379:methods* / Q000639:trends*; D014161:Transduction, Genetic / Q000379:methods* / Q000639:trends
chemical_list: C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator
keywords: CFTR gene; cystic fibrosis; gene therapy; prenatal