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.

selected publications
Academic Articles16
  • Nghiem, P. P., & Kornegay, J. N. (2019). Correction to: Gene therapies in canine models for Duchenne muscular dystrophy. 138(5), 491-491.
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  • Nghiem, P. P., & Kornegay, J. N. (2019). Gene therapies in canine models for Duchenne muscular dystrophy. 138(5), 483-489.
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  • Guo, L., Soslow, J. H., Bettis, A. K., Nghiem, P. P., Cummings, K. J., Lenox, M. W., ... Spurney, C. F. (2019). Natural History of Cardiomyopathy in Adult Dogs With Golden Retriever Muscular Dystrophy.. J Am Heart Assoc. (16),
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  • Mata Lpez, S., Hammond, J. J., Rigsby, M. B., Balog-Alvarez, C. J., Kornegay, J. N., & Nghiem, P. P. (2018). A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20.. Skelet Muscle. (1),
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  • Schneider, S. M., Sridhar, V., Bettis, A. K., Heath-Barnett, H., Balog-Alvarez, C. J., Guo, L., ... Nghiem, P. P. (2018). Glucose Metabolism as a Pre-clinical Biomarker for the Golden Retriever Model of Duchenne Muscular Dystrophy.. Mol Imaging Biol. (5),
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  • Nghiem, P. P., & Schatzberg, S. (2012). Acute blindness. Small Animal Neurological Emergencies. 229-242.
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  • Schatzberg, S., & Nghiem, P. P. (2012). Infectious and inflammatory diseases of the CNS. Small Animal Neurological Emergencies. 341-362.
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Conference Papers1
  • 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. 25(5), 193-193.
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Texas A&M University; Veterinary Integrative Biosciences; 4458 TAMU
College Station, TX 77843-4458