Mark A. Vanden Avond

Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice

Shelby E. Hamm, Daniel D. Fathalikhani, Katherine E. Bukovec et al. · Molecular Therapy — Methods & Clinical Development · 2021 · 24 citations · Full text

Medicine, Molecular Genetics, Physiology +14

Aberrations in Energetic Metabolism and Stress-Related Pathways Contribute to Pathophysiology in the Neb Conditional Knockout Mouse Model of Nemaline Myopathy

Rebecca A. Slick, Jennifer A Tinklenberg, J. R. Sutton et al. · American Journal Of Pathology · 2023 · 12 citations · Full text

Life Sciences, Molecular Biology, Molecular Genetics +17

Different Mouse Models of Nemaline Myopathy Harboring Acta1 Mutations Display Differing Abnormalities Related to Mitochondrial Biology

Jennifer A Tinklenberg, Rebecca A. Slick, J. R. Sutton et al. · American Journal Of Pathology · 2023 · 11 citations · Full text

Life Sciences, Genetics, Molecular Genetics +14

The nucleotide prodrug <scp>CERC</scp>‐913 improves <scp>mtDNA</scp> content in primary hepatocytes from <scp>DGUOK‐deficient</scp> rats

Mark A. Vanden Avond, Hui Meng, Margaret Beatka et al. · Journal of Inherited Metabolic Disease · 2020 · 10 citations

Life Sciences, Cell Biology, Molecular Biology +15

Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial

Michael W. Lawlor, Benedikt Schoser, Marta Margeta et al. · EBioMedicine · 2023 · 10 citations · Full text

Medicine, Molecular Genetics, Molecular Medicine +14