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Updated: Jul 28, 2026

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
Published on: November 25, 2012
Perspectives in gene therapy for MS
1Department of Neuroscience-DIBIT, San Raffaele Scientific Institute, Via Olgettina 58, 20132 Milan, Italy. g.martino@hsr.it
Abstract:
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.
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