Investigator: Yi-Wen Chen DMV PhD
Category: Research - Translational
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
Grant Information
Date awarded: May 13, 2017
End date: May 12, 2018
Amount awarded: $78,798
Children's Research Institute
Yi-Wen Chen DMV PhD
Associate Professor
Children's Research Institute
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