Technology evaluation: AAV-CFTR vector, targeted genetics.

  • S. J. Tebbutt
  • Published 1999 in Current opinion in molecular therapeutics


Targeted Genetics is developing a gene therapy product, the AAV-CFTR vector system, for the treatment of cystic fibrosis (CF). This involves administration of an adeno-associated virus (AAV) vector containing CF transmembrane conductance regulator gene (CFTR) directly to the lungs of cystic fibrosis patients. The drug has Orphan Drug Status [325713]. Medeva… (More)