Inventions, Patents and Portfolios - WARF
Technologies

Pharmaceuticals & Vitamin D : Orphan diseases

Technologies

Method and Compounds for Treating Friedreich’s Ataxia

UW–Madison researchers have developed a chimeric synthetic molecule capable of inducing increases in FXN mRNA levels in Friedreich’s ataxia patient cell lines.

The complex has the formula A-L-B wherein A- is a bromodomain inhibitor, -L- is a linker, and -B is a polyamide that specifically binds to one or more repeats of a GAA oligonucleotide sequence. Mechanistically, when the complex binds to the repeat sequence, the bromodomain inhibitor (ex., Brd4) recruits the superelongation complex (SEC) to restart the paused transcription complex.
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