Synthetic transcriptional activators (syn-TFs): a new therapeutic platform for the treatment of Friedrich's Ataxia (FRDA)

合成转录激活剂（syn-TF）：治疗弗里德里希共济失调（FRDA）的新治疗平台

• 批准号: 2280115
• 金额: --
• 依托单位: University of Strathclyde
• 依托单位国家: 英国
• 项目类别: Studentship
• 财政年份: 2019
• 资助国家: 英国
• 起止时间: 2019 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=studentship-2280115
• 关键词: Synthetic; transcriptional; activators; syn; TFs

One of the holy grails of genetic medicine is to develop therapies which can correct aberrant gene expression. This is particularly challenging with genetic diseases such as Friedrich's Ataxia (FRDA). FRDA arises when repeating GAA-TTC sequences of DNA build up in the frataxin gene (FXN), which results in a significant reduction in the production of frataxin. The overall objective of this studentship is to develop a new generation of chimeric molecules which can activate FXN transcription. The studentship will involve a blend of synthetic chemistry, chemical biological probe design, and compound optimization guided by deep-sequencing analysis. This project directly aligns with the EPSRC's Chemical Biology & Biological Chemistry research area in the Healthcare Technologies Theme.

基因医学的圣杯之一是开发出能够纠正异常基因表达的疗法。这对于遗传性疾病如弗里德里希共济失调（FRDA）尤其具有挑战性。当重复的GAA-TTC DNA序列在共济失调蛋白基因（FXN）中积累时，FRDA就会出现，这导致共济失调蛋白的产生显著减少。本研究的总体目标是开发新一代能够激活FXN转录的嵌合分子。该奖学金将涉及合成化学，化学生物探针设计和深度测序分析指导的化合物优化的混合物。该项目直接与EPSRC在医疗技术主题中的化学生物学和生物化学研究领域保持一致。