US trial of gene therapy for cystic fibrosisBMJ 1999; 318 doi: https://doi.org/10.1136/bmj.318.7191.1096 (Published 24 April 1999) Cite this as: BMJ 1999;318:1096
- Janice Hopkins Tanne
- New York
Researchers at Stanford University Medical Center in California have started a trial of gene therapy delivered by aerosol to the lungs of patients with cystic fibrosis. The treatment consists of a manufactured version of the faulty cystic fibrosis gene packed inside the shell of a virus.
“We use a manufactured version of the cystic fibrosis transmembrane conductance regulator (CFTR) gene packed into an adeno-associated virus shell. The adeno-associated virus is a small, single stranded RNA virus. It …