Study design (if review, criteria of inclusion for studies)
double-blinded, placebo-controlled, clinical study
Participants
12 CF patients: 8 received treatment, 4 received placebo
Interventions
Cationic liposomes complexed with plasmid containing the human CFTR cDNA were administered to patients
Outcome measures
Biopsies of the nasal epithelium taken 7 days after dosing were normal
Main results
No significant changes in clinical parameters were observed. Functional expression of CFTR assessed by in vivo nasal potential difference measurements showed transient correction of the CF chloride transport abnormality in two patients (15 days after dosing in one patient). Fluorescence microscopy demonstrated CFTR function ex vivo. In cells from nasal brushings. In total, evidence of functional CFTR gene transfer was obtained in six out of the eight treated patients
Authors' conclusions
These results provide proof of concept for liposome-mediated CF gene transfer.
Keywords: Adolescent; Adult; Gene Transfer Techniques; non pharmacological intervention - genetic& reprod; pharmacological_intervention; placebo;