Scholars

Associate Professor

Positions:

Associate Professor, Veterinary Integrative Biosciences, School of Veterinary Medicine and Biomedical Sciences

overview

Molecular, cellular, and phenotypic characterization of the canine models for Duchenne muscular dystrophy (golden retriever muscular dystrophy [GRMD]; german short-haired pointer muscular dystrophy [GSHPMD]; cavalier king charles spaniel muscular dystrophy). Molecular characterization with genome-wide mRNA and microRNA profiling via Affymetrix chip and proteomic profiling with mass spectrometry. Confirmation of molecular targets with qRT-PCR, western blot, immunofluorescence microscopy, etc. Cellular characterization of the canine models utilizing biopsy extracted muscle stem cells (myoblasts), including evaluation of the molecular and phenotypic effects of various treatments. Phenotypic characterization of the canine models using internationally established functional outcome measures developed in the Kornegay laboratory. Current research focus is on preclinical drug trials, including gene therapy (dystrophin gene replacement) via adeno-associated viral vector delivery; utilzing gene editing techniques such as CRISPR/Cas9 and TALENs for treatments of genetic disease; characterization of genetic modifiers via whole-genome next generation sequencing (discovery approach); and evaluation of muscle metabolism in dystrophin deficiency.

education and training

Ph.D. in Molecular Medicine, George Washington University - (Washington D.C., District of Columbia, United States) 2014
D.V.M. in Medicine & Surgery, Texas A&M University - (College Station, Texas, United States) 2008

selected publications

Academic Articles26

Russell, A. J., DuVall, M., Barthel, B., Qian, Y., Peter, A. K., Newell-Stamper, B. L., ... Koch, K. (2023). Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy.. Journal of Clinical Investigation. 133(10), e153837.
Birch, S. M., Lawlor, M. W., Conlon, T. J., Guo, L., Crudele, J. M., Hawkins, E. C., ... Kornegay, J. N. (2023). Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.. Science Translational Medicine. 15(677), eabo1815.
Rutledge, A. M., Guo, L., Lord, L. E., Leal, A. R., Deramus, J., Lpez, S. M., Russell, A., & Nghiem, P. P. (2022). Comprehensive assessment of physical activity correlated with muscle function in canine Duchenne muscular dystrophy.. Annals of Physical and Rehabilitation Medicine. 65(5), 101611-101611.
Martin, P. T., Zygmunt, D. A., Ashbrook, A., Hamilton, S., Packer, D., Birch, S. M., ... Kornegay, J. N. (2021). Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength.. 16(3), e0248721-e0248721.
Lpez, S. M., Balog-Alvarez, C., Canessa, E. H., Hathout, Y., Brown, K. J., Vitha, S., ... Nghiem, P. P. (2020). Creation and characterization of an immortalized canine myoblast cell line: Myok9.. Mammalian Genome. 31(3-4), 95-109.

Chapters2

Nghiem, P. P., & Schatzberg, S. (2012). Acute blindness. Small Animal Neurological Emergencies. 229-242.
Schatzberg, S., & Nghiem, P. P. (2012). Infectious and inflammatory diseases of the CNS. Small Animal Neurological Emergencies. 341-362.

Conference Papers2

Uranga, S., O'Reilly, C. L., Lewis, M. H., Lopez, S. M., Nghiem, P. P., & Fluckey, J. D. (2022). Anabolic Characterization Of Immortalized Murine L6, And Canine Myok9 Skeletal Myotubes. Medicine & Science in Sports & Exercise. 54(9), 391-391.
Birch, S. M., Lawlor, M. W., Guo, L., Crudele, J. M., Hawkins, E. C., Nghiem, P. P., ... Kornegay, J. N. (2017). A Blinded, Placebo-Controlled Systemic Gene Therapy Efficacy Study in the GRMD Model of Duchenne Muscular Dystrophy. Molecular Therapy. 25(5), 193-193.