Airway gene transfer in a non-human primate: Lentiviral gene expression in marmoset lungs

N. Farrow, D. Miller, P. Cmielewski, M. Donnelley, R. Bright, D. W. Parsons

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23 Citations (Scopus)
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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.

Original languageEnglish
Article number1287
Number of pages4
JournalScientific Reports
Volume3
DOIs
Publication statusPublished - 15 Feb 2013
Externally publishedYes

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