Systemic antisense therapeutics inhibiting DUX4 expression improves muscle function in an FSHD mouse model
Ngoc Lu‐Nguyen, Alberto Malerba, Shan Herath, George Dickson, Linda J. Popplewell
This study shows that systemic antisense therapy targeting DUX4 improves muscle function in an FSHD mouse model by reducing DUX4 expression and associated pathology.
Human Molecular Genetics · May 11, 2021Peer-reviewedMolecular Biology