caa a22 4500 475748522 CHVBK 20180406123520.0 cr unu---uuuuu 170329e20000901xx s 000 0 eng 10.1023/A:1006616029205 doi (NATIONALLICENCE)springer-10.1023/A:1006616029205 Gene Therapy for Autoimmune Disorders [Elektronische Daten] [C. Evans, S. Ghivizzani, T. Oligino, P. Robbins] Although many autoimmune disorders do not have a strong genetic basis, their treatment may nevertheless be improved by gene therapies. Most strategies seek to transfer genes encoding immunomodulatory products that will alter host immune responses in a beneficial manner. Used in this fashion, genes serve as biological delivery vehicles for the products they encode. By this means gene therapy overcomes obstacles to the targeted delivery of proteins and RNA, and improves their efficacy while providing a longer duration of effect, and, potentially, greater safety. Additional genetic strategies include DNA vaccination and the ablation of selected tissues and cell populations. There is considerable evidence from animal studies that gene therapies work: examples include the treatment of experimental models of rheumatoid arthritis, multiple sclerosis, diabetes, and lupus. Pre-clinical success in treating animal models of rheumatoid arthritis has led to the first clinical trial of gene therapy for an autoimmune disease. In this Phase I study, a cDNA encoding the interleukin-1 receptor antagonist was transferred to the knuckle joints of patients with advanced rheumatoid arthritis. Two additional clinical trials are in progress. It is likely that gene therapy will provide effective new treatments for a wide range of autoimmune disorders. Plenum Publishing Corporation, 2000 Gene nationallicence vector nationallicence cytokine nationallicence synovium nationallicence dendritic cell nationallicence T-lymphocyte nationallicence human trial nationallicence Evans C. Center for Molecular Orthopaedics, Department of Orthopaedic Surgery, Harvard Medical School, 02115, Boston, Massachusetts aut Ghivizzani S. Center for Molecular Orthopaedics, Department of Orthopaedic Surgery, Harvard Medical School, 02115, Boston, Massachusetts aut Oligino T. Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania aut Robbins P. Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania aut Journal of Clinical Immunology Kluwer Academic Publishers-Plenum Publishers 20/5(2000-09-01), 334-346 0271-9142 20:5<334 2000 20 10875 https://doi.org/10.1023/A:1006616029205 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1023/A:1006616029205 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Evans C. Center for Molecular Orthopaedics, Department of Orthopaedic Surgery, Harvard Medical School, 02115, Boston, Massachusetts aut NATIONALLICENCE 700 1- Ghivizzani S. Center for Molecular Orthopaedics, Department of Orthopaedic Surgery, Harvard Medical School, 02115, Boston, Massachusetts aut NATIONALLICENCE 700 1- Oligino T. Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania aut NATIONALLICENCE 700 1- Robbins P. Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania aut NATIONALLICENCE 773 0- Journal of Clinical Immunology Kluwer Academic Publishers-Plenum Publishers 20/5(2000-09-01), 334-346 0271-9142 20:5<334 2000 20 10875 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer