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.

引用

页数：4

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