snoRNA Vectors for Sequence-specific Gene Knockdown in human cells

用于人类细胞中序列特异性基因敲除的 snoRNA 载体

• 批准号: G0801738/1
• 负责人: Angus Lamond
• 金额: $ 65.76万
• 依托单位: University of Dundee
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2009
• 资助国家: 英国
• 起止时间: 2009 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G0801738%2F1
• 关键词: snoRNA; Vectors; Sequence; specific; Gene

Genes carry the genetic instructions to allow cells to build proteins that control chemical reactions within cells and that regulate cell growth and division. Mutations can cause genes to make defective proteins that in turn cause many forms of human disease, including cancer, diabetes and inherited disorders. This proposal will develop a new technology that allows researchers to control which genes are active in human cells and also allows them to inactivate the defective copies of mutated genes and replace them with new genes that can now make the correct and active form of the same protein. This can be widely used to help test and develop new drugs and may also be developed as a direct therapy for use in patients.

基因携带遗传指令，允许细胞构建控制细胞内化学反应并调节细胞生长和分裂的蛋白质。突变会导致基因产生有缺陷的蛋白质，进而导致多种形式的人类疾病，包括癌症、糖尿病和遗传性疾病。这项提案将开发一种新技术，使研究人员能够控制哪些基因在人类细胞中是活跃的，并使他们能够消除突变基因的缺陷拷贝，并用新的基因取代它们，这些基因现在可以使相同蛋白质的正确和活跃形式。这可以广泛用于帮助测试和开发新药，也可以开发为用于患者的直接疗法。