pGT-1 gene lipid complex

**pGT-1 gene lipid complex** is an investigational nonviral **CFTR gene-replacement therapy** for cystic fibrosis. It consists of pGT-1 DNA encoding a cystic fibrosis gene copy formulated with the cationic liposome components DMRIE/DOPE to promote uptake by nasal epithelial cells. In the University of Alabama at Birmingham-sponsored Phase 1 study, the lipid/DNA formulation was instilled by syringe into the right inferior nasal turbinate under direct visualization over 30 minutes to assess delivery of the cystic fibrosis gene to nasal-lining cells. ([clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00004471))

Other names

pGT-1 lipid complexpGT1 lipid complexpGT 1 lipid complex