{"title":"多囊肾病的新治疗方法","authors":"Ameya Patil, W. Sweeney, C. Pan, E. Avner","doi":"10.3233/TRD-190040","DOIUrl":null,"url":null,"abstract":"Polycystic kidney disease (PKD) and nephronophthisis are common manifestation of ciliopathies. PKD is the most common genetic renal condition; it affects 12.5 million people worldwide. PKD is a great example of decades of translational research leading to the discovery of novel treatments and significant number of clinical trials. This review will concentrate on the basic molecular and cellular pathophysiology that led to the development of therapeutic targets for PKD.","PeriodicalId":75246,"journal":{"name":"Translational science of rare diseases","volume":"74 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.3233/TRD-190040","citationCount":"2","resultStr":"{\"title\":\"Novel Treatments for Polycystic Kidney Disease\",\"authors\":\"Ameya Patil, W. Sweeney, C. Pan, E. Avner\",\"doi\":\"10.3233/TRD-190040\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Polycystic kidney disease (PKD) and nephronophthisis are common manifestation of ciliopathies. PKD is the most common genetic renal condition; it affects 12.5 million people worldwide. PKD is a great example of decades of translational research leading to the discovery of novel treatments and significant number of clinical trials. This review will concentrate on the basic molecular and cellular pathophysiology that led to the development of therapeutic targets for PKD.\",\"PeriodicalId\":75246,\"journal\":{\"name\":\"Translational science of rare diseases\",\"volume\":\"74 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2019-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.3233/TRD-190040\",\"citationCount\":\"2\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Translational science of rare diseases\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.3233/TRD-190040\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Translational science of rare diseases","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.3233/TRD-190040","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Polycystic kidney disease (PKD) and nephronophthisis are common manifestation of ciliopathies. PKD is the most common genetic renal condition; it affects 12.5 million people worldwide. PKD is a great example of decades of translational research leading to the discovery of novel treatments and significant number of clinical trials. This review will concentrate on the basic molecular and cellular pathophysiology that led to the development of therapeutic targets for PKD.
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