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

被引：21
作者：
Farrow, N. [1 ,2 ,3 ]
Miller, D. [1 ,4 ]
Cmielewski, P. [1 ,2 ,3 ]
Donnelley, M. [1 ,2 ,3 ]
Bright, R. [1 ,4 ]
Parsons, D. W. [1 ,2 ,3 ,4 ]
机构：
[1] Womens & Childrens Hlth Network, Adelaide, SA 5006, Australia
[2] Univ Adelaide, Ctr Stem Cell Res, Adelaide, SA 5000, Australia
[3] Univ Adelaide, Dept Paediat & Reprod Hlth, Adelaide, SA 5000, Australia
[4] Womens & Childrens Hlth Res Inst, Adelaide, SA 5000, Australia
来源：
SCIENTIFIC REPORTS | 2013年 / 3卷
基金：
澳大利亚国家健康与医学研究理事会;
关键词：
CYSTIC-FIBROSIS; MOUSE TRACHEA; STEM-CELLS; VECTOR; THERAPY; MICE;
D O I：
10.1038/srep01287
中图分类号：
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号：
07 ; 0710 ; 09 ;
摘要：
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.
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页数：4
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