University of California, Davis (UCD) is exploring the use of Artificial Transcription Factors/Zinc Fingers (ATF-ZF) as an approach to unsilence the paternal UBE3A gene as a treatment strategy for Angelman syndrome.
A therapeutic approach that consists of using engineered proteins designed to regulate gene expression in a highly specific manner. They are constructed to bind to specific DNA sequences and can either activate or repress the expression of target genes. ATF-Zinc fingers are small proteins that use zinc ions to stabilize their structures giving them a finger-like appearance. ATF-ZFs can bind to the UBE3A-ATS and prevent the silencing of the paternal copy of the UBE3A gene.
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