The use of a recombinant lentiviral vector for ex vivo gene transfer into the rat CNS

A major obstacle in ex vivo gene transfer has been the loss of transgene ex pression soon after implantation of the grafted transduced cells. Recently, a lentiviral vector system has been developed which has proven to express high levels of transgenes in vivo after direct injection into the tissue. I n this study, we have investigated the use of such a vector for ex vivo gen e transfer to the brain. A number of neural cell types were found to be per missive to transduction by the lentiviral vector in vitro and a majority of them expressed the transgene after transplantation to the rat brain. Trans gene expression was detected up to 8 weeks post-grafting. These findings su ggest that recombinant lentiviral vectors may be used for further developme nt of ex vivo gene therapy protocols to the CNS. NeuroReport 11:3973-3977 ( C) 2000 Lippincott Williams & Wilkins.