Affordable Access

Publisher Website

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

Authors
Journal
Scientific Reports
2045-2322
Publisher
Nature Publishing Group
Publication Date
Volume
3
Identifiers
DOI: 10.1038/srep01287
Keywords
  • Article
Disciplines
  • Biology
  • Medicine

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.

There are no comments yet on this publication. Be the first to share your thoughts.