Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes.

ABSTRACT: Gene therapy for cystic fibrosis (CF) is making encouraging progress into clinical trials. However, further improvements in transduction efficiency are desired. To develop a novel gene transfer vector that is improved and truly effective for CF gene therapy, a simian immunodeficiency virus (SIV) was pseudotyped with envelope proteins from Sendai virus (SeV), which is known to efficiently transduce unconditioned airway epithelial cells from the apical side. This novel vector was evaluated in mice in vivo and in vitro directed toward CF gene therapy. Here, we show that (i) we can produce relevant titers of an SIV vector pseudotyped with SeV envelope proteins for in vivo use, (ii) this vector can transduce the respiratory epithelium of the murine nose in vivo at levels that may be relevant fo

SUBMITTER: Mitomo K 

PROVIDER: S-EPMC2889732 | biostudies-literature | 2010 Jun

REPOSITORIES: biostudies-literature