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.
- 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
- Nghiem, P. P., Bello, L., Stoughton, W. B., López, S. M., Vidal, A. H., Hernandez, B. V., ... Kornegay, J. N. (2017). Changes in Muscle Metabolism are Associated with Phenotypic Variability in Golden Retriever Muscular Dystrophy.. YALE JOURNAL OF BIOLOGY AND MEDICINE. 90(3), 351-360.
- Nghiem, P. P., Kornegay, J. N., Uaesoontrachoon, K., Bello, L., Yin, Y., Kesari, A., ... Hoffman, E. P. (2017). Osteopontin is linked with AKT, FoxO1, and myostatin in skeletal muscle cells. Muscle & Nerve. 56(6), 1119-1127.
- Nghiem, P. P., Bello, L., Balog-Alvarez, C., López, S. M., Bettis, A., Barnett, H., ... Kornegay, J. N. (2016). Whole genome sequencing reveals a 7 base-pair deletion in DMD exon 42 in a dog with muscular dystrophy. Mammalian Genome. 28(3-4), 106-113.
- Acosta, A. R., Van Wie, E., Stoughton, W. B., Bettis, A. K., Barnett, H. H., LaBrie, N. R., ... Kornegay, J. N. (2016). Use of the six-minute walk test to characterize golden retriever muscular dystrophy. Neuromuscular Disorders. 26(12), 865-872.
- Many, G. M., Yokosaki, Y., Uaesoontrachoon, K., Nghiem, P. P., Bello, L., Dadgar, S., ... Hoffman, E. P. (2016). OPN‐a induces muscle inflammation by increasing recruitment and activation of pro‐inflammatory macrophages. Experimental Physiology. 101(10), 1285-1300.
- 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.