Cystic fibrosis gene therapy.
A variety of cystic fibrosis gene therapy approaches based on viral (adenovirus, retrovirus, and adeno-associated virus) and non-viral (liposomes and receptor-mediated endocytosis) routes are currently being assessed for safety and efficacy. Of these, the trials involving liposomal and adenoviral vectors are the most advanced, as both have been shown to correct the cystic fibrosis Cl- conductance defect in vivo.
Colledge
W H
WH
Wellcome/CRC Institute of Cancer and Developmental Biology, Cambridge, UK.
D000260:Adenoviruses, Human / Q000235:genetics; D000818:Animals; D018118:Chloride Channels / Q000235:genetics; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D018076:DNA, Complementary / Q000008:administration & dosage / Q000235:genetics; D000229:Dependovirus / Q000235:genetics; D004195:Disease Models, Animal; D004705:Endocytosis; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008081:Liposomes; D008565:Membrane Proteins / Q000235:genetics; D012190:Retroviridae / Q000235:genetics
C505032:CFTR protein, human; D018118:Chloride Channels; D018076:DNA, Complementary; D008081:Liposomes; D008565:Membrane Proteins; D019005:Cystic Fibrosis Transmembrane Conductance Regulator