{"title":"光遗传学治疗对多发性硬化症患者的治疗潜力","authors":"Mohamed M. El Koussy, N. Jadavji","doi":"10.22186/jyi.33.4.77-82","DOIUrl":null,"url":null,"abstract":"been found (Harbo, Gold, & Tintoré, 2013). Genetically, MS is best characterized by a mutation on the human leukocyte antigen (HLA) gene locus, which causes abnormal antigen recognition of T cells leading to attacks on myelin proteins (Raffel et al., 2016). These findings have not been conclusive, as many other genes involved in immunological roles have also been found to play a role in contributing to MS. Environmental risk factors include smoking ,sunlight exposure, and vitamin D deficiency (Raffel et al., 2016). Currently, there are no treatments that cure MS (Ziemssen et al., 2016). Instead, treatments target symptom management to increase patients’ quality of life. These include high doses of corticosteroids such as methylprednisolone (Jongen et al., 2016), Disease modifying Treatments (DMTs) such as interferon β-1a, interferon β-1b, alemtuzumab, fingolimod and natalizumab (Carrithers et al., 2014; Gajofatto & Benedetti, 2015), and neuro-rehabilitation (Dasari, Wootla, Warrington, & Rodriguez, 2016). All of these treatment options have adverse effects or are not particularly effective in the long term (Jongen et al., 2016; Ontaneda, Fox, & Chataway, 2015; Schäcke, Döcke, & Asadullah, 2002; Ziemssen et al., 2016). There is a considerable need for new treatment options that are more effective, while reducing the adverse side-effects. A potential therapy for MS-affected individuals may be the therapeutic application of optogenetics. Optogenetics is a novel method that utilizes photoreceptors to selectively activate neurons (Hegemann & Nagel, 2013). The genetic code of these receptors is delivered either virally or nonvirally to be expressed on the cells of interest. Once expressed, Introduction Multiple Sclerosis (MS) is a chronic autoimmune disease that leads to focal and diffuse neurodegenration and myelination throughout the nervous system (Kolasinski et al., 2012; Siffrin, Vogt, Radbruch, Nitsch, & Zipp, 2010). In its most common form, relapseremitting MS, it is characterized by high inflammation levels that lead to a continuous cycle of relapse and remission (Raffel, Wakerley, & Nicholas, 2016). These relapses, called exacerbations, may come in the form of new or worsening of old symptoms that are largely neurological such as visual impairment and imbalance that worsen over days or weeks, then recover spontaneously (Wingerchuk et al., 2014). Other common symptoms are cognitive impairment, loss of bladder control, leg weakness and sensory symptoms (Raffel et al., 2016). Genetic and environmental factors both have a role in MS development; however, a specific link to the disease has not Therapeutic Potential of Optogenetic Treatment for Individuals with Multiple Sclerosis","PeriodicalId":74021,"journal":{"name":"Journal of young investigators","volume":" ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2017-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":"{\"title\":\"Therapeutic potential of optogenetic treatment for individuals with multiple sclerosis\",\"authors\":\"Mohamed M. El Koussy, N. Jadavji\",\"doi\":\"10.22186/jyi.33.4.77-82\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"been found (Harbo, Gold, & Tintoré, 2013). Genetically, MS is best characterized by a mutation on the human leukocyte antigen (HLA) gene locus, which causes abnormal antigen recognition of T cells leading to attacks on myelin proteins (Raffel et al., 2016). These findings have not been conclusive, as many other genes involved in immunological roles have also been found to play a role in contributing to MS. Environmental risk factors include smoking ,sunlight exposure, and vitamin D deficiency (Raffel et al., 2016). Currently, there are no treatments that cure MS (Ziemssen et al., 2016). Instead, treatments target symptom management to increase patients’ quality of life. These include high doses of corticosteroids such as methylprednisolone (Jongen et al., 2016), Disease modifying Treatments (DMTs) such as interferon β-1a, interferon β-1b, alemtuzumab, fingolimod and natalizumab (Carrithers et al., 2014; Gajofatto & Benedetti, 2015), and neuro-rehabilitation (Dasari, Wootla, Warrington, & Rodriguez, 2016). All of these treatment options have adverse effects or are not particularly effective in the long term (Jongen et al., 2016; Ontaneda, Fox, & Chataway, 2015; Schäcke, Döcke, & Asadullah, 2002; Ziemssen et al., 2016). There is a considerable need for new treatment options that are more effective, while reducing the adverse side-effects. A potential therapy for MS-affected individuals may be the therapeutic application of optogenetics. Optogenetics is a novel method that utilizes photoreceptors to selectively activate neurons (Hegemann & Nagel, 2013). The genetic code of these receptors is delivered either virally or nonvirally to be expressed on the cells of interest. Once expressed, Introduction Multiple Sclerosis (MS) is a chronic autoimmune disease that leads to focal and diffuse neurodegenration and myelination throughout the nervous system (Kolasinski et al., 2012; Siffrin, Vogt, Radbruch, Nitsch, & Zipp, 2010). In its most common form, relapseremitting MS, it is characterized by high inflammation levels that lead to a continuous cycle of relapse and remission (Raffel, Wakerley, & Nicholas, 2016). These relapses, called exacerbations, may come in the form of new or worsening of old symptoms that are largely neurological such as visual impairment and imbalance that worsen over days or weeks, then recover spontaneously (Wingerchuk et al., 2014). Other common symptoms are cognitive impairment, loss of bladder control, leg weakness and sensory symptoms (Raffel et al., 2016). Genetic and environmental factors both have a role in MS development; however, a specific link to the disease has not Therapeutic Potential of Optogenetic Treatment for Individuals with Multiple Sclerosis\",\"PeriodicalId\":74021,\"journal\":{\"name\":\"Journal of young investigators\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2017-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of young investigators\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.22186/jyi.33.4.77-82\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of young investigators","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.22186/jyi.33.4.77-82","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Therapeutic potential of optogenetic treatment for individuals with multiple sclerosis
been found (Harbo, Gold, & Tintoré, 2013). Genetically, MS is best characterized by a mutation on the human leukocyte antigen (HLA) gene locus, which causes abnormal antigen recognition of T cells leading to attacks on myelin proteins (Raffel et al., 2016). These findings have not been conclusive, as many other genes involved in immunological roles have also been found to play a role in contributing to MS. Environmental risk factors include smoking ,sunlight exposure, and vitamin D deficiency (Raffel et al., 2016). Currently, there are no treatments that cure MS (Ziemssen et al., 2016). Instead, treatments target symptom management to increase patients’ quality of life. These include high doses of corticosteroids such as methylprednisolone (Jongen et al., 2016), Disease modifying Treatments (DMTs) such as interferon β-1a, interferon β-1b, alemtuzumab, fingolimod and natalizumab (Carrithers et al., 2014; Gajofatto & Benedetti, 2015), and neuro-rehabilitation (Dasari, Wootla, Warrington, & Rodriguez, 2016). All of these treatment options have adverse effects or are not particularly effective in the long term (Jongen et al., 2016; Ontaneda, Fox, & Chataway, 2015; Schäcke, Döcke, & Asadullah, 2002; Ziemssen et al., 2016). There is a considerable need for new treatment options that are more effective, while reducing the adverse side-effects. A potential therapy for MS-affected individuals may be the therapeutic application of optogenetics. Optogenetics is a novel method that utilizes photoreceptors to selectively activate neurons (Hegemann & Nagel, 2013). The genetic code of these receptors is delivered either virally or nonvirally to be expressed on the cells of interest. Once expressed, Introduction Multiple Sclerosis (MS) is a chronic autoimmune disease that leads to focal and diffuse neurodegenration and myelination throughout the nervous system (Kolasinski et al., 2012; Siffrin, Vogt, Radbruch, Nitsch, & Zipp, 2010). In its most common form, relapseremitting MS, it is characterized by high inflammation levels that lead to a continuous cycle of relapse and remission (Raffel, Wakerley, & Nicholas, 2016). These relapses, called exacerbations, may come in the form of new or worsening of old symptoms that are largely neurological such as visual impairment and imbalance that worsen over days or weeks, then recover spontaneously (Wingerchuk et al., 2014). Other common symptoms are cognitive impairment, loss of bladder control, leg weakness and sensory symptoms (Raffel et al., 2016). Genetic and environmental factors both have a role in MS development; however, a specific link to the disease has not Therapeutic Potential of Optogenetic Treatment for Individuals with Multiple Sclerosis