Novel Therapies for Cystinosis

胱氨酸病的新疗法

• 批准号: 312156
• 负责人: Goodyer Paul R
• 金额: $ 16.39万
• 依托单位: Research Institute of the McGill University Health Centre
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2014
• 资助国家: 加拿大
• 起止时间: 2014-05-01 至 2017-05-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=312156&lang=en
• 关键词: Cystinosis; Drug Library Screening; Mutant Mouse Model; Mutant Zebrafish Model; Renal Progenitor Cell

Cystinosis is a rare genetic disease causing kidney failure in childhood and progressive deterioration of other organs later in early adulthood. Current therapies are inadequate; although disease progression is slowed, all cystinosis patients require kidn

胱氨酸病是一种罕见的遗传性疾病，在儿童期引起肾衰竭，在成年早期引起其他器官的进行性退化。目前的治疗是不够的，虽然疾病进展减缓，所有胱氨酸病患者需要肾脏治疗。