snoRNA Vectors for Sequence-specific Gene Knockdown in human cells

基因携带遗传指令，使细胞能够构建蛋白质，控制细胞内的化学反应，调节细胞的生长和分裂。突变可以导致基因产生缺陷蛋白，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。这项提议将开发一种新技术，使研究人员能够控制哪些基因在人类细胞中是活跃的，并允许他们使突变基因的缺陷拷贝失活，并用现在可以制造相同蛋白质的正确和活跃形式的新基因来取代它们。这可以广泛用于帮助测试和开发新药，也可以开发为患者使用的直接疗法。

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.