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Conclusions The gene therapy of autoimmunity has held many promises for the last ten years, owing to its potential as an alternative therapeutic approach for diseases lacking a definitive cure and with a devastating social impact. However, there are still several issues to solve before these approaches would be transferable to humans. Some studies are conceptually non applicable to human diseases...
Conclusion While pharmacologic agents will no doubt continue to be discovered to promote safer immunosuppression, insulin sensitisation and enhancement of insulin output, diabetes mellitus will continue to be a challenging disorder in which these agents could be applied. Gene therapeutics, however, will take advantage of the knowledge of the underlying stage and severity of diabetes and will very...
Conclusions The majority of gene therapy studies have been performed with viral vectors that present important limitations in terms of immunogenicity and pathogenicity. Nonviral (usually plasmid-based) gene therapy is not hampered by these limitations and, although gene transfer is generally less efficient, it has been successfully employed in the pre-vention or treatment of several experimental autoimmune...
One of the most exciting advances in the field of gene therapy in recent years is the establishment of the antigen-specific T cell as a vector for the delivery of genetically-derived treatment in vivo. In contrast with traditional applications of gene therapy, the unique versatility, specificity and memory of the T cell affords the researcher or clinician the ability to apply a broad range of tactics...
Conclusions In RA, which is both a polyarticular and a systemic disease, anti-TNF or anti-IL-1 tar geted treatment will not be the only solutions proposed to patients in the future. Gene therapy approaches, when introduced, do not imply the exclusion of former therapies. They are only specific and promising examples of targeted therapies that are not based on the use of protein anymore, but rather...
Conclusions The mechanisms of tolerance induction and its breakdown are important to explore because of its involvement in the pathogenesis of many known autoimmune diseases. Tolerance to “self” is not absolute and can be overcome by the immune system after a foreign stimuli caused by pathogens, allergens or other imknown immune errors (e.g., defects in apoptosis) that affect the immune system, causing...
Conclusions The ultimate goal in the treatment of autoimmune diseases is to restore immune tolerance to the relevant target antigen(s). Short of this ideal, the attenuation of pathogenic immune responses is a highly desirable end. Many forms of immunotherapy are being studied with these objectives in mind, but gene transfer approaches, and particularly DNA vaccination (transfer of an antigen gene),...
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