RNA-seq of murine tibialis anterior muscle treated with AAV expressing AR isoform 2 (AR45) or AAV-GFP

The goal of this study was to determine the transcriptomic response of AR-2 (AR45) overexpression in the TA muscles of the AR100Q murine model of SBMA (spinal and bulbar muscular atrophy). Conclusion: AAV-AR45 therapy resulted in a partial restoration of the SBMA-like expression signature compared with mock-treated controls. RNA-seq was performed on tibialis anterior muscles from SBMA (spinal and bulbar muscular atrophy) AR100Q mice treated with AAV vectors expressing either AR-2 (AR45) or GFP mock control. Untreated wild-type mice (C57BL/6) were analysed in parallel as additional controls.