Muscle-targeted complexes and their use for treating facial, scapular, and brachial muscular dystrophy

摘要

Aspects of the present disclosure relate to complexes comprising muscle targeting agents covalently linked to a molecular payload. In some embodiments, the muscle targeting agent specifically binds to internalized cell surface receptors on muscle cells. In some embodiments, the molecular payload inhibits DUX4 expression or activity. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or an RNAi oligonucleotide.