AAV Gene Therapy to Modify Promoter Function

Researchers at Nationwide Children’s Hospital developed a new technology to regulate gene expression. They propose utilizing U7 small nuclear RNA with a tail will attract endogenous activator or pressor proteins. The U7 small RNA will guide RNAs to bind to promoter sequences to alter gene expression by blocking activator or repressor elements in promoters. The attached tail will consist of specific binding sequences recognized by the repressor or activator proteins attracting them to the intended promoter for its regulation.

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