Perspectives in gene therapy for MS.
The blood-brain barrier limits the therapeutic efficacy of systemic administration of anti-inflammatory and/or neuroprotective molecules to patients affected by immune-mediated inflammatory demyelinating diseases of the central nervous system (CNS) such as multiple sclerosis. Drug delivery to the CNS using non-replicative viral vectors may represent a valid alternative therapeutic strategy. Gene therapy for multiple sclerosis might include different "human-grade" vectors, which could be used to deliver anti-inflammatory molecules as well as neuroprotective agents into the CNS in a flexible and useful way. These potential "therapeutic" vectors would have different life spans, tissue tropism and infectivity rates.
Martino
G
G
Department of Neuroscience-DIBIT, San Raffaele Scientific Institute, Via Olgettina 58, 20132 Milan, Italy. g.martino@hsr.it
D005544:Forecasting; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D006801:Humans; D009103:Multiple Sclerosis / Q000628:therapy*