snoRNA Vectors for Sequence-specific Gene Knockdown in human cells

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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.