9 research outputs found
Evaluation of promoters for driving efficient transgene expression in neonatal porcine islets
A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery
In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA
Treatment of multifocal breast cancer by systemic delivery of dual-targeted adeno-associated viral vectors
Transgene and islet cell delivery systems using nano-sized carriers for the treatment of diabetes mellitus
Gene therapy for autoimmune diseases: quo vadis?
Biological therapies using antibodies and cytokines are becoming widespread for the treatment of chronic inflammatory autoimmune diseases. However, these treatments have several limitations - such as expense, the need for repeated injections and unwanted side-effects - that can be overcome by genetic delivery. This review summarizes the ingenuity, sophistication and variety of gene-therapy approaches that have been taken in the design of therapeutic molecules and vectors, the engineering of cells and the regulation of gene expression for the targeting of disease outcome. We focus our attention on multiple sclerosis, type 1 diabetes and rheumatoid arthritis.Fil: Chernajovsky, Yuti. University of London; Reino UnidoFil: Gould, David J.. University of London; Reino UnidoFil: Podhajcer, Osvaldo Luis. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Parque Centenario. Instituto de Investigaciones Bioquímicas de Buenos Aires. Fundación Instituto Leloir. Instituto de Investigaciones Bioquímicas de Buenos Aires; Argentin