Lentiviral vectors as tools to model and treat neurodegenerative diseases:
The identification of disease-causing genes in familial forms of neurodegenerative disorders has drastically changed our understanding of the molecular events leading to neuronal cell death. These achievements open new opportunities for the development of genetic models closely replicating human CNS pathologies and therapeutic interventions by gene therapy. However, the presence of the blood-brain barrier and the post-mitotic and poor regenerative nature of the target cells constitute important challenges. Efficient gene transfer systems taking into account the specificity of the central nervous system are required to develop pertinant models of neurodegenerative diseases and administer potential therapeutic candidates.
Third generation tat-dependent HIV-1 based lentiviral vectors suitable for
the silencing of target genes with siRNA or the overexpression of reporter,
disease or therapeutic genes are produced for in vitro and in vivo applications