Generation of transgenic mice using lentiviral vectors: a novel preclinical assessment of lentiviral vectors for gene therapy

摘要: Lentiviral vectors have become attractive delivery vehicles for gene therapy investigators. Specifically, the ability of lentiviral to integrate into nondividing cells and provide stable long-term expression in vivo is a desirable attribute approaches. We report here simple method generating transgenic mice using vectors, which could be useful models therapy. After removal zona pellucida, fertilized eggs were co-incubated with oncoretroviral or vectors. The resulting blastocysts transferred uteri pseudo-pregnant females. In both cases, around 60–70% founder pups as determined by PCR analysis. Southern blot analysis revealed that transgenes integrated at different genetic loci transmitted through germ line. Most delivered expressed mice, although those completely silenced. When upstream sequences rhodopsin red pigment used tissue-specific promoters, consistent enhanced green fluorescent protein (EGFP) was observed rod cone photoreceptor cells, respectively, retina. However, generated corneal epithelium-specific keratin-12 promoter displayed EGFP not only cornea but also other tissues mouse. conclude generation robust evaluate specificity prior undertaking strategy.