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

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.

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