Gene Therapy Approaches in an Autoimmune Demyelinating Disease: Multiple Sclerosis.

作者: Md. A. Islam , Shoumik Kundu , Rosline Hassan

DOI: 10.2174/1566523220666200306092556

关键词: ImmunologyAutoimmune diseaseMedicineDemyelinating diseaseAutoimmunityMultiple sclerosisRemyelinationCentral nervous systemExperimental autoimmune encephalomyelitisGenetic enhancement

摘要: Multiple Sclerosis (MS) is the most common autoimmune demyelinating disease of Central Nervous System (CNS). It a multifactorial which develops in an immune-mediated way under influences both genetic and environmental factors. Demyelination observed brain spinal cord leading to neuro-axonal damage patients with MS. Due infiltration different immune cells such as T-cells, B-cells, monocytes macrophages, focal lesions are Currently available medications treating MS mainly based on two strategies; i) ease specific symptoms or ii) reduce progression. However, these tend induce adverse effects limited therapeutic efficacy due protective function blood-brain barrier. Therefore, researchers have been working for last four decades discover better solutions by introducing gene therapy approaches generally following three strategies, prevention symptoms, halt reverse progression iii) heal CNS promoting remyelination axonal repair. In decades, there some remarkable successes experimental mice model - encephalomyelitis (EAE) suggests that it not far would start human subjects ensuring highest levels safety efficacy. this review, we summarised attempted animal models towards

参考文章(94)
W.W. Hauswirth, X. Qi, J. Guy, L. Sun, AAV-Mediated Sod2 Gene Expression Driven by a Pro-Inflammatory Inducible Promoter: A Novel Method for Gene Therapy of Multiple Sclerosis Investigative Ophthalmology & Visual Science. ,vol. 44, pp. 628- 628 ,(2003)
Alemtuzumab vs. interferon beta-1a in early multiple sclerosis. The New England Journal of Medicine. ,vol. 359, pp. 1786- 1801 ,(2008) , 10.1056/NEJMOA0802670
Calliope A. Dendrou, Lars Fugger, Manuel A. Friese, Immunopathology of multiple sclerosis Nature Reviews Immunology. ,vol. 15, pp. 545- 558 ,(2015) , 10.1038/NRI3871
Francisco J Quintana, Mauricio F Farez, Soledad Pérez-Sánchez, Inmunopatología de la esclerosis múltiple. Medicina-buenos Aires. ,vol. 74, pp. 404- 410 ,(2014)
R Furlan, A Bergami, E Brambilla, E Butti, M G De Simoni, M Campagnoli, P Marconi, G Comi, G Martino, HSV-1-mediated IL-1 receptor antagonist gene therapy ameliorates MOG(35-55)-induced experimental autoimmune encephalomyelitis in C57BL/6 mice. Gene Therapy. ,vol. 14, pp. 93- 98 ,(2007) , 10.1038/SJ.GT.3302805
Adil Javed, Anthony T. Reder, Therapeutic role of beta-interferons in multiple sclerosis. Pharmacology & Therapeutics. ,vol. 110, pp. 35- 56 ,(2006) , 10.1016/J.PHARMTHERA.2005.08.011
Amit Bar-Or, Andrew Pachner, Francoise Menguy-Vacheron, Johanne Kaplan, Heinz Wiendl, Teriflunomide and Its Mechanism of Action in Multiple Sclerosis Drugs. ,vol. 74, pp. 659- 674 ,(2014) , 10.1007/S40265-014-0212-X
Suhayl Dhib-Jalbut, David Trisler, Tapas K. Makar, Christopher T. Bever, Ishwar S. Singh, Walter Royal, Surasri Nandan Sahu, Tushar P. Sura, Shireen Sultana, Karna T. Sura, Niraj Patel, Brain-derived neurotrophic factor gene delivery in an animal model of multiple sclerosis using bone marrow stem cells as a vehicle. Journal of Neuroimmunology. ,vol. 210, pp. 40- 51 ,(2009) , 10.1016/J.JNEUROIM.2009.02.017
D. Ganea, M. Toscano, F. Emig, K. Hooper, 48. Neuropeptides as cell gene therapy in experimental autoimmune encephalomyelitis Brain Behavior and Immunity. ,vol. 32, ,(2013) , 10.1016/J.BBI.2013.07.060