RNA sequencing of control, non-treated FSHD, and LNA gapmer-treated FSHD myotubes. Homo sapiens

Immortalized myoblasts derived from a facioscapulohumeral muscular dystrophy (FSHD) patient and a healthy subject were grown and differentiated. FSHD patient-derived cells were either treated or not treated with an LNA gapmer targeting knockdown of the DUX4 transcript. RNA sequencing was performed on total RNA extracts from these three groups (healthy, FSHD non-treated, and FSHD treated) to evaluate the efficacy of LNA gapmer treatment at the transcriptomic level.