Publications
2023
Novel Metabolomic Approach for Identifying Pathology-Specific Biomarkers in Rare Diseases: A Case Study in Oculopharyngeal Muscular Dystrophy (OPMD)
Harish, P., Malerba, A., Kroon, R. H. M. J. M., Shademan, M., van Engelan, B., Raz, V., . . . Snowden, S. G. G. (2023). Novel Metabolomic Approach for Identifying Pathology-Specific Biomarkers in Rare Diseases: A Case Study in Oculopharyngeal Muscular Dystrophy (OPMD). METABOLITES, 13(6). doi:10.3390/metabo13060769
Systemic Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the A17 Mouse Model of OPMD.
Malerba, A., Harish, P., & Popplewell, L. (2023). Systemic Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the A17 Mouse Model of OPMD.. In Unknown Book (Vol. 2587, pp. 557-568). doi:10.1007/978-1-0716-2772-3_30
2021
Knockdown of Muscle-Specific Ribosomal Protein L3-Like Enhances Muscle Function in Healthy and Dystrophic Mice
Kao, B. R., Malerba, A., Lu-Nguyen, N. B., Harish, P., McCarthy, J. J., Dickson, G., & Popplewell, L. J. (2021). Knockdown of Muscle-Specific Ribosomal Protein L3-Like Enhances Muscle Function in Healthy and Dystrophic Mice. NUCLEIC ACID THERAPEUTICS, 31(6), 457-464. doi:10.1089/nat.2020.0928
2020
Age-Associated Salivary MicroRNA Biomarkers for Oculopharyngeal Muscular Dystrophy
Raz, V., Kroon, R. H. M. J. M., Mei, H., Riaz, M., Buermans, H., Lassche, S., . . . van Engelen, B. G. M. (2020). Age-Associated Salivary MicroRNA Biomarkers for Oculopharyngeal Muscular Dystrophy. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 21(17). doi:10.3390/ijms21176059
Fast Facts: Gene Therapy
Herzog, R., & Popplewell, L. (2020). Fast Facts: Gene Therapy. doi:10.1159/isbn.978-3-318-06667-8
Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease
Harish, P., Forrest, L., Herath, S., Dickson, G., Malerba, A., & Popplewell, L. (2020). Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease. FRONTIERS IN PHYSIOLOGY, 11. doi:10.3389/fphys.2020.00184
2019
Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)
Harish, P., Malerba, A., Ngoc, L. -N., Forrest, L., Cappellari, O., Roth, F., . . . Dickson, G. (2019). Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD). JOURNAL OF CACHEXIA SARCOPENIA AND MUSCLE, 10(5), 1016-1026. doi:10.1002/jcsm.12438
A pilot study to elucidate effects of artificial selection by size on the zebrafish (<i>Danio rerio</i>) fast skeletal muscle transcriptome
Harish, P., Mareco, E. A., & de la Serrana, G. D. (2019). A pilot study to elucidate effects of artificial selection by size on the zebrafish (<i>Danio rerio</i>) fast skeletal muscle transcriptome. COMPARATIVE BIOCHEMISTRY AND PHYSIOLOGY A-MOLECULAR & INTEGRATIVE PHYSIOLOGY, 233, 65-73. doi:10.1016/j.cbpa.2019.03.021
Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy
Malerba, A., Roth, F., Harish, P., Dhiab, J., Ngoc, L. -N., Cappellari, O., . . . Trollet, C. (2019). Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy. HUMAN MOLECULAR GENETICS, 28(10), 1694-1708. doi:10.1093/hmg/ddz007
Gene Therapy for Oculopharyngeal Muscular Dystrophy
Malerba, A., Roth, F., Strings, V., Harish, P., Suhy, D., Trollet, C., & Dickson, G. (2019). Gene Therapy for Oculopharyngeal Muscular Dystrophy. In Muscle Gene Therapy (pp. 549-564). Springer International Publishing. doi:10.1007/978-3-030-03095-7_31
2018
Advances in emerging therapeutics for oculopharyngeal muscular dystrophy
Harish, P., Dickson, G., & Malerba, A. (2018). Advances in emerging therapeutics for oculopharyngeal muscular dystrophy. EXPERT OPINION ON ORPHAN DRUGS, 6(11), 693-701. doi:10.1080/21678707.2018.1536542
2017
Nuclear PABPN1 aggregates in OPMD: correlation study and therapy
Roth, F., Jarmin, S., Oliver, A., Nguyen, N., Chappell, A., Harish, P., . . . Trollet, C. (2017). Nuclear PABPN1 aggregates in OPMD: correlation study and therapy. NEUROMUSCULAR DISORDERS, 27, S202-S203. doi:10.1016/j.nmd.2017.06.394
2015
Progress on Gene Therapy, Cell Therapy, and Pharmacological Strategies Toward the Treatment of Oculopharyngeal Muscular Dystrophy
Harish, P., Malerba, A., Dickson, G., & Bachtarzi, H. (2015). Progress on Gene Therapy, Cell Therapy, and Pharmacological Strategies Toward the Treatment of Oculopharyngeal Muscular Dystrophy. HUMAN GENE THERAPY, 26(5), 286-292. doi:10.1089/hum.2015.014