Treating Facioscapulohumeral Muscular Dystrophy

SLU ID 13-019 | Small molecule inhibition of BET bromodomain proteins

Background

Facioscapulohumeral Muscular Dystrophy (FSHD) is characterized by epigenetic changes resulting in the aberrant expression of the DUX4 gene in muscle. Suppression of DUX4 expression is considered a therapeutic target for FSHD. However, the mechanisms responsible for DUX expression are poorly understood and few targets amenable to small molecule drug intervention have been described.

Overview

Researchers at Saint Louis University have identified inhibitors of BET bromodomain proteins that block induction of DUX4 expression and activity.

Benefits

The potential benefits of this technology include:

• Increasing the potential treatments for FSHD

• Minimizing muscle degeneration in FSHD patients

Applications

The potential applications of this technology include:

• Developing treatments for Facioscapulohumeral Muscular Dystrophy (FSHD)

Opportunity

Saint Louis University is seeking a partner to further develop and commercialize this technology.