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CFTR gene-liposome complex
Dosage:
- every 4 weeks by nebulizer [1]
Mechanism of action:
- contains normal working copy of CFTR gene delivered in liposomal complex by nebulizer [1]
General
aerosolized agent
References
- Alton EW, Armstrong DK, Ashby D et al
Repeated nebulisation of non-viral CFTR gene therapy in
patients with cystic fibrosis: a randomised, double-blind,
placebo-controlled, phase 2b trial.
Lancet. July 3, 2015
PMID: 26149841
http://www.thelancet.com/pdfs/journals/lanres/PIIS2213-2600%2815%2900245-3.pdf