friends of FSH Research


Investigation of a third generation oligonucleotide targeting DUX4 using a new mouse model of FSHD

Investigator: Yi-Wen Chen DMV PhD

Facioscapulohumeral muscular dystrophy (FSHD) is a rare disease caused by aberrant expression of double homeobox 4 (DUX4) in patients’ muscles. Reducing the DUX4 expression in the muscles is an acute area of research since it is directly relevant to disease treatment. The goal of this study is to evaluate a promising strategy, called third generation antisense (3GA) developed by the Idera Pharmaceuticals using a novel mouse model of FSHD. We will determine whether the treatment of a 3GA targeting DUX4 improve muscle health and functions. In addition, we will evaluate the safety of the 3GA by checking changes in muscles and blood samples.

Related Material

Progress Report

Grant Information

Date awarded: May 13, 2017
End date: May 13, 2018
Amount awarded: $78,798

Yi-Wen Chen DMV PhD
Associate Professor
Children's Research Institute