Transcriptional profiling of Gastrocnemius Muscle in Pompe's disease

Study identifying transcriptional differences present between C57BL/6J and B6;129-Gaatm1Rabn/J mice, or driven by Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), or Both, administered to the mutant strain. All expression profiles were assessed in bulk gastrocnemius muscle collected from 18-21 week-old mice that had been provided with chow formulated with vehicle (veh.), MZ-101 (SRT), or alglucosidase alfa 20mg/kg biweekly (ERT) for 12 weeks. Overall design: Comparative gene expression profiling analysis of RNA-seq data derived from mouse models of Pompe's disease treated with vehicle, SRT, ERT, or Combo, versus wildtype strain