Correction of Friedreich ataxia induced pluripotent stem cells by non-viral gene therapy

非病毒基因治疗纠正 Friedreich 共济失调诱导的多能干细胞

• 批准号: nhmrc : 1055785
• 负责人: Miss Tejal Kulkarni
• 金额: $ 4.22万
• 依托单位: Murdoch Childrens Research Institute
• 依托单位国家: 澳大利亚
• 项目类别: Postgraduate Scholarships
• 财政年份: 2014
• 资助国家: 澳大利亚
• 起止时间: 2014-01-01 至 2016-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/correction-friedreich-ataxia-gene-therapy/112492
• 关键词: Correction; Friedreich; ataxia; induced; pluripotent

Friedreich ataxia (FRDA) is an inherited progressive disorder of the nervous system and heart. Stem cell therapy has the potential to repair or replace damaged tissues and restore organ function in FRDA patients. The defect inherent in stem cells obtained from FRDA patients will be corrected by a gene therapy approach that will restore normal FRDA gene expression and addresses major safety concerns for the clinical use of corrected stem cells in transplantation medicine.

弗里德赖希共济失调 (FRDA) 是一种遗传性进行性神经系统和心脏疾病。干细胞疗法有可能修复或替换 FRDA 患者受损的组织并恢复器官功能。从 FRDA 患者获得的干细胞固有的缺陷将通过基因治疗方法得到纠正，该方法将恢复正常的 FRDA 基因表达，并解决移植医学中临床使用纠正的干细胞的主要安全问题。