{"title":"一种治疗亨廷顿舞蹈病的新疗法。","authors":"Albert R La Spada","doi":"","DOIUrl":null,"url":null,"abstract":"<p><p><i>In 1979, while at the National Institutes of Health, now Columbia University professor Nancy Wexler and colleagues traveled to Venezuela to study the world's largest family with Huntington's disease. That led to identifying the disease gene at the tip of human chromosome 4 and the race to find a drug that can treat people who carry the fatal gene prior to the onset of symptoms. Our author believes that a new strategy tied to turning off targeted genes could have profound implications for therapy development for Huntington's and other neurodegenerative diseases</i>.</p>","PeriodicalId":72553,"journal":{"name":"Cerebrum : the Dana forum on brain science","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2018-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6353115/pdf/","citationCount":"0","resultStr":"{\"title\":\"A Novel Therapy for Huntington's Disease.\",\"authors\":\"Albert R La Spada\",\"doi\":\"\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p><i>In 1979, while at the National Institutes of Health, now Columbia University professor Nancy Wexler and colleagues traveled to Venezuela to study the world's largest family with Huntington's disease. That led to identifying the disease gene at the tip of human chromosome 4 and the race to find a drug that can treat people who carry the fatal gene prior to the onset of symptoms. Our author believes that a new strategy tied to turning off targeted genes could have profound implications for therapy development for Huntington's and other neurodegenerative diseases</i>.</p>\",\"PeriodicalId\":72553,\"journal\":{\"name\":\"Cerebrum : the Dana forum on brain science\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2018-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6353115/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cerebrum : the Dana forum on brain science\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cerebrum : the Dana forum on brain science","FirstCategoryId":"1085","ListUrlMain":"","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
1979年，在美国国立卫生研究院(National Institutes of Health)工作期间，现哥伦比亚大学(Columbia University)教授南希·韦克斯勒(Nancy Wexler)和同事前往委内瑞拉，研究世界上最大的亨廷顿舞蹈病家族。这导致了人类4号染色体尖端的疾病基因的识别，以及寻找一种可以在症状出现之前治疗携带致命基因的人的药物的竞赛。我们的作者认为，一种与关闭目标基因相关的新策略可能对亨廷顿舞蹈症和其他神经退行性疾病的治疗发展产生深远的影响。
In 1979, while at the National Institutes of Health, now Columbia University professor Nancy Wexler and colleagues traveled to Venezuela to study the world's largest family with Huntington's disease. That led to identifying the disease gene at the tip of human chromosome 4 and the race to find a drug that can treat people who carry the fatal gene prior to the onset of symptoms. Our author believes that a new strategy tied to turning off targeted genes could have profound implications for therapy development for Huntington's and other neurodegenerative diseases.
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