AAV-U7snRNA to Modulate Promoter Activity for Myotonic Dystrophy

Myotonic dystrophy can occur at any age when there are mutations to the DMPK gene which causes patients to experience muscle weakness throughout the body. Researchers at Nationwide Children’s Hospital developed new technology to modulate the promoter activity to alter the DMPK gene expression by using U7 snRNA with a tail encoded with specific binding sequences. The tail will attract endogenous activator/repressor proteins which naturally exist in mammalian cells and bind to DNA sequences or secondary tissue. The binding of the protein to a promoter will allow for promoter regulation to activate or repress promoters; therefore, upregulating or downregulating gene expression of a certain gene like the DMPK gene.

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