Targeting Autoimmune and Neurological Disease by Gene Therapy

gene therapy

The laboratory of Nathan Karin investigates the molecular basis of autoimmune inflammatory processes, particularly within in the central nervous system (CNS). To accomplish this task the lab uses experimental models, in rodents, for Multiple Sclerosis (MS) in comparison with models of Rheumatoid Arthritis (RA). Nathan Karin and his team has demonstrated that during these autoimmune inflammatory conditions the immune system mounts a significant response against a few mediators that participate in the regulation of each autoimmune condition. Targeted DNA vaccines encoding several of these mediators may elicit protective immunity. A biotechnology company that has been established within the Rappaport Institute (Vaccinogen) will explore the therapeutic potential of these findings. In another complementary pathway the lab inserts genes of interest to myelin basic protein specific T cells and uses them as a vehicle to transfer genes of interest to the brain. In the near future the lab intends to use this sophisticated strategy to suggest novel therapeutic strategies for MS and possibly other CNS inflammatory diseases such as Alzheimer’s disease.

Representative Publications:

Wildbaum G., Youssef S. and Karin N. (2000) A targeted DNA vaccine augments the natural immune response to self TNF-a and suppresses adjuvant arthritis J. Immunol.; 165, 5860-5866.

Wildbaum G., Westerman J., Maor G., and Karin N. (2000) A targeted DNA vaccine induces the breakdown of tolerance to Fas ligand and explores its dual role in experimental autoimmune encephalomyelitis. J. Clin. Invest.; 106, 671-679.

Youssef S., Maor G., Grabie N., Wildbaum G., Gour-Lavie A., and Karin N. (2000) C-C chemokine encoding DNA vaccines inhibit ongoing adjuvant arthritis. J. Clin. Invest.; 106, 361-371.


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