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• W2035133781 abstract "Genetic therapies for cystic fibrosis (CF) must be assessed for safety and efficacy, so testing in a non-human primate (NHP) model is invaluable. In this pilot study we determined if the conducting airways of marmosets (n = 2) could be transduced using an airway pre-treatment followed by an intratracheal bolus dose of a VSV-G pseudotyped HIV-1 based lentiviral (LV) vector (LacZ reporter). LacZ gene expression (X-gal) was assessed after 7 days and found primarily in conducting airway epithelia as well as in alveolar regions. The LacZ gene was not detected in liver or spleen via qPCR. Vector p24 protein bio-distribution into blood was transient. Dosing was well tolerated. This preliminary study confirmed the transducibility of CF-relevant airway cell types. The marmoset is a promising NHP model for testing and translating genetic treatments for CF airway disease towards clinical trials." @default.
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• W2035133781 date "2013-02-15" @default.
• W2035133781 modified "2023-09-25" @default.
• W2035133781 title "Airway gene transfer in a non-human primate: Lentiviral gene expression in marmoset lungs" @default.
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• W2035133781 doi "https://doi.org/10.1038/srep01287" @default.
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