Direct injection of plasmid DNA into the brain

Publisher Summary The cationic lipid-mediated DNA delivery into the brain, without the use of viral infection, has several advantages. The construction of plasmid DNAs containing functional genes and adequate enhancers/promoters is easily performed, and is less time consuming than generating viral vectors. The use of plasmid DNAs to deliver genetic materials is not associated with risks of deleterious side effects, such as cytotoxicity, autoimmune encephalitis, or productive virus infection. When the β-galactosidase gene is fused with the nuclear location signal derived from the SV40 T-antigen gene as with the plasmid L7RHβ-gal, the β-galactosidase protein produced can be transported into the nuclei of cells. The possible utilization of expression plasmid vectors as DNA delivery vehicles for gene transfer into rodent brains was initially demonstrated with neonatal mice. It is suggested that the transfection efficiency of direct gene transfer be raised for obtaining reproducible effects on the recovery of functionally deficient phenotypes.