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., & Kornegay, J. N. (2019). Gene therapies in canine models for Duchenne muscular dystrophy. Hum Genet. 138(5), 483-489.
- Nghiem, P. P., & Kornegay, J. N. (2019). Gene therapies in canine models for Duchenne muscular dystrophy (vol 138, pg 483, 2019). Hum Genet. 138(5), 491-491.
- 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. Journal of the American Heart Association. 8(16), e012443.
- Lopez, S. M., 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. SKELETAL MUSCLE. 8(1), 16.
- 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. Molecular Imaging and Biology. 20(5), 780-788.
- 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.