{"title":"A Conversation with Adrian Krainer.","authors":"","doi":"10.1101/sqb.2019.84.039461","DOIUrl":null,"url":null,"abstract":"Anke Sparmann: You were awarded the 2019 Breakthrough Prize in Life Sciences together with Dr. Frank Bennett of Ionis Pharmaceuticals for the development of antisense oligonucleotide drugs to target RNA splicing and the incredible success story of SPINRAZA, the first drug approved for spinal muscular atrophy. Can you start by telling us about this devastating disease and the molecular mechanism underlying it that you discovered?","PeriodicalId":72635,"journal":{"name":"Cold Spring Harbor symposia on quantitative biology","volume":"84 ","pages":"276-278"},"PeriodicalIF":0.0000,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1101/sqb.2019.84.039461","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cold Spring Harbor symposia on quantitative biology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1101/sqb.2019.84.039461","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2020/3/13 0:00:00","PubModel":"Epub","JCR":"","JCRName":"","Score":null,"Total":0}
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Abstract
Anke Sparmann: You were awarded the 2019 Breakthrough Prize in Life Sciences together with Dr. Frank Bennett of Ionis Pharmaceuticals for the development of antisense oligonucleotide drugs to target RNA splicing and the incredible success story of SPINRAZA, the first drug approved for spinal muscular atrophy. Can you start by telling us about this devastating disease and the molecular mechanism underlying it that you discovered?
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