Adeno-associated virus and lentivirus pseudotypes for lung-directed gene therapy.
The enthusiasm for cystic fibrosis gene therapy that attended the initial cloning of the gene and in vitro correction of the genetic defect eventually diminished as we learned more about the limitations of vector technologies that were available in the 1980s and 1990s. Substantial progress has been made, however, over the last 5 years in developing second- and third-generation vector constructs that should be more useful in achieving gene transfer to the lung for the treatment of pulmonary diseases such as cystic fibrosis.
Wilson
James M
JM
204 Wistar Institute, 3601 Spruce Street, Philadelphia, PA 19104-4268, USA. wilsonjm@mail.med.upenn.edu
D000256:Adenoviridae / Q000235:genetics*; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage*; D006801:Humans; D016086:Lentivirus / Q000235:genetics*; D012680:Sensitivity and Specificity; D016896:Treatment Outcome