Please use this identifier to cite or link to this item:
https://hdl.handle.net/2440/78539
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Type: | Journal article |
Title: | Airway gene transfer in a non-human primate: Lentiviral gene expression in marmoset lungs |
Author: | Farrow, N. Miller, D. Cmielewski, P. Donnelley, M. Bright, R. Parsons, D. |
Citation: | Scientific Reports, 2013; 3(1):1-3 |
Publisher: | Nature Group |
Issue Date: | 2013 |
ISSN: | 2045-2322 2045-2322 |
Statement of Responsibility: | N. Farrow, D. Miller, P. Cmielewski, M. Donnelley, R. Bright and D.W. Parsons |
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 5 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. |
Keywords: | Genetic vectors; preclinical research; stem-cell research; transfection |
Rights: | This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/3.0/ |
DOI: | 10.1038/srep01287 |
Grant ID: | NHMRC |
Published version: | http://dx.doi.org/10.1038/srep01287 |
Appears in Collections: | Aurora harvest 4 Paediatrics publications |
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hdl_78539.pdf | Published version | 553.42 kB | Adobe PDF | View/Open |
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