Development of single-stranded RNAsfor therapeutic gene knock-down

用于治疗性基因敲低的单链RNA的开发

• 批准号: 2271216
• 金额: --
• 依托单位: University of Nottingham
• 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-2271216
• 关键词: Development; single; stranded; RNAsfor; therapeutic

This project will study key features of the triplet repeat RNA sequence associated with myotonic dystrophy type 1 and type 2. Specifically we will:1. Perform Super Resolution microscopy to confirm or refute the presence of CUG repeat fragments in the nuclear and cytoplasmic compartments of DM1 cell lines.2. Examine DM2 cell lines for similar fragments of CCUG repeats.3. Perform oligo-based capture experiments with subsequent analysis (TLC + Mass Spec) to determine whether fragments, if present, are subjected to base modification. 4. Use CRISPR Cas 9 to generate DM cell lines with inducible CUG and CCUG repeats.

该项目将研究与1型和2型强直性肌营养不良相关的三重重复RNA序列的关键特征。具体来说，我们将：1。进行超分辨率显微镜检查，以确认或反驳DM1细胞系的细胞核和细胞质区室中存在CUG重复片段。检查DM2细胞系中CCUG重复序列的相似片段。进行基于寡核苷酸的捕获实验，并进行后续分析（TLC +质谱），以确定片段（如果存在）是否经过碱基修饰。4.使用CRISPR Cas 9生成具有诱导型CUG和CCUG重复序列的DM细胞系。