Applications of gene therapy to the CNS.
Gene therapy is a new method with potential for treating a broad range of acquired and inherited neurologic diseases, where the causative gene defect or deletion has been identified. In addition to gene replacement the application of gene products that reduce cellular dysfunction or death represent new therapeutic options. Gene transfer techniques to express novel proteins using different viral vectors in vitro and in vivo, as well as animal models and human trials will be reviewed in this article. We will focus on a new lentiviral vector as a recent gene transfer method and degenerative disorders of the CNS, and their related model systems.
Blömer
U
U
Salk Institute for Biological Studies, La Jolla, CA 92037-1099, USA.
Naldini
L
L
Verma
I M
IM
Trono
D
D
Gage
F H
FH
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D002490:Central Nervous System / Q000378:metabolism*; D000229:Dependovirus / Q000235:genetics; D004195:Disease Models, Animal; D005544:Forecasting; D015316:Genetic Therapy; D005822:Genetic Vectors; D006564:Herpesviridae / Q000235:genetics; D006801:Humans; D009474:Neurons / Q000378:metabolism; D012190:Retroviridae / Q000235:genetics