{"title":"一种新的左旋多巴反应性肌张力障碍小鼠模型的实用性评论。","authors":"Samuel J Rose, Ellen J Hess","doi":"10.1080/21675511.2015.1128617","DOIUrl":null,"url":null,"abstract":"<p><p>In a recent issue of Brain, we reported on the generation and characterization of a mouse model of the rare disease L-DOPA-responsive dystonia (DRD). Here, we discuss the utility of these mice for understanding broader disease processes and treatment strategies. Using specific experimental designs that either work \"forward\" from genetic etiology or \"backward\" from the symptomatic presentation, we discuss how our data and future work can be used to understand broader themes. </p>","PeriodicalId":74639,"journal":{"name":"Rare diseases (Austin, Tex.)","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2015-12-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21675511.2015.1128617","citationCount":"0","resultStr":"{\"title\":\"A commentary on the utility of a new L-DOPA-responsive dystonia mouse model.\",\"authors\":\"Samuel J Rose, Ellen J Hess\",\"doi\":\"10.1080/21675511.2015.1128617\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>In a recent issue of Brain, we reported on the generation and characterization of a mouse model of the rare disease L-DOPA-responsive dystonia (DRD). Here, we discuss the utility of these mice for understanding broader disease processes and treatment strategies. Using specific experimental designs that either work \\\"forward\\\" from genetic etiology or \\\"backward\\\" from the symptomatic presentation, we discuss how our data and future work can be used to understand broader themes. </p>\",\"PeriodicalId\":74639,\"journal\":{\"name\":\"Rare diseases (Austin, Tex.)\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2015-12-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1080/21675511.2015.1128617\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Rare diseases (Austin, Tex.)\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1080/21675511.2015.1128617\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2016/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Rare diseases (Austin, Tex.)","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1080/21675511.2015.1128617","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2016/1/1 0:00:00","PubModel":"eCollection","JCR":"","JCRName":"","Score":null,"Total":0}
A commentary on the utility of a new L-DOPA-responsive dystonia mouse model.
In a recent issue of Brain, we reported on the generation and characterization of a mouse model of the rare disease L-DOPA-responsive dystonia (DRD). Here, we discuss the utility of these mice for understanding broader disease processes and treatment strategies. Using specific experimental designs that either work "forward" from genetic etiology or "backward" from the symptomatic presentation, we discuss how our data and future work can be used to understand broader themes.
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