The development of flexible lentiviral vectors for gene transfer in the CNS

Abstract

The use of recombinant lentiviral vectors (rLV) is emerging as a viable candidate for clinical gene therapy of the central nervous system. New generation vectors are being produced while addressing viral safety concerns as well as production capabilities. Furthermore, the ability to combine envelope proteins targeting specific cell types with specific promoters guiding the expression of the genetic payload will allow researchers and clinicians to precisely guide transgene expression to anatomically and phenotypically distinct populations of cells. In a recent issue of Experimental Neurology, Cannon and colleagues demonstrate the ability to transduce specific populations of cells in the rat midbrain by using differently pseudotyped lentiviral vectors which results in significant differences in transgene spread throughout the nigrostriatal tract. These results highlight the potential utility of rLV in clinical applications as well as in research involving neurodegenerative disease.