Emily Ott

The Effect of Immunomodulatory Treatments on Anti-Dystrophin Immune Response After AAV Gene Therapy in Dystrophin Deficient mdx Mice

Ning Li, Joanna E. Parkes, Rita Spathis et al. · Journal of Neuromuscular Diseases · 2021 · 15 citations

Medicine, Immunology, Pathobiology +17

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

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