Gene therapy in the treatment of autoimmune disease

✍ Scribed by Peter M. Mathisen; Vincent K. Tuohy

• Publisher: Elsevier Science
• Year: 1998
• Tongue: English
• Weight: 323 KB
• Volume: 19
• Category: Article
• ISSN: 0167-5699
• DOI: 10.1016/s0167-5699(98)80005-4

✦ Synopsis

he in vivo local delivery of regulatory cytokines made possible by gene therapy is becoming an exciting approach for treating common disorders such as cancer and viral infections I. Recently, gene therapy strategies have been applied to the treatment of autoimmune diseases. Cells targeted for autoimmune attack may be genetically modified to express immunoregulatory cytokines that protect them from autoimmune-mediated destruction (target tissue gene therapy). Such target cells include pancreatic [~ cells in type I insulin-dependent diabetes mellitus (IDDM), myelin-producing oligodendrocytes in multiple sclerosis (MS), and synoviocytes in rheumatoid arthritis (RA). In a complementary strategy, autoreactive T cells may be genetically altered to deliver anti-inflammatory cytokines to autoimmune lesions (T-cellmediated gene therapy). In addition, autoimmune target cells and autoreactive T cells may be genetically modified to produce therapeutic transgene growth factors for mediating repair of tissue damaged during autoimmune disease (regenerative gene therapy). The proposed strategies for gene therapy treatment of autoimmune disease are depicted in Fig. 1.