AAV-mediated gene transfer restores a normal muscle transcriptome in a canine model of X-linked myotubular myopathy
J.-B. Dupont, J. Guo, M. W. Lawlor, R. W. Grange, J. T. Gray, A. Buj-Bello, M. K. Childers, D. L. Mack
The paper uses RNA-sequencing to analyze the transcriptome of canine XLMTM muscle after rAAV8-cMTM1 gene transfer, showing dose-dependent normalization of gene expression and identifying new disease-related transcripts.
bioRxiv · Dec 17, 2018Pathology