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.

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