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

doi:10.1038/srep01287

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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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 language	English
Article number	1287
Number of pages	4
Journal	Scientific Reports
Volume	3
DOIs	https://doi.org/10.1038/srep01287
Publication status	Published - 15 Feb 2013
Externally published	Yes

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AU - Miller, D.

AU - Cmielewski, P.

AU - Donnelley, M.

AU - Bright, R.

AU - Parsons, D. W.

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

Abstract

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