# positions[].organizations[].parent[].label
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.

# keywords[]
Positron Emission Tomography Computed Tomography

# keywords[]
4.1 Discovery And Preclinical Testing Of Markers And Technologies

# keywords[]
Intercellular Signaling Peptides And Proteins

# educationAndTraining[].organization.label
George Washington University - (Washington D.C., District of Columbia, United States)

# educationAndTraining[].organization.label
Texas A&M University - (College Station, Texas, United States)

# publications[].label
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.

# publications[].label
Changes in Muscle Metabolism are Associated with Phenotypic Variability in Golden Retriever Muscular Dystrophy.

# publications[].label
Glucose Metabolism as a Pre-clinical Biomarker for the Golden Retriever Model of Duchenne Muscular Dystrophy.

# publications[].label
Conventional and molecular diagnostic testing for the acute neurologic patient.

# publications[].label
A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20.

# publications[].label
The weak cat. Practical approach and common neurological differentials.

# publications[].label
Correction to: Gene therapies in canine models for Duchenne muscular dystrophy.

# publications[].label
Comprehensive assessment of physical activity correlated with muscle function in canine Duchenne muscular dystrophy.

# publications[].label
Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies.

# publications[].label
Creation and characterization of an immortalized canine myoblast cell line: Myok9.

# publications[].label
Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMDgenetic mutation in canine Duchenne muscular dystrophy.

# publications[].label
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.

# publications[].label
The paradox of muscle hypertrophy in muscular dystrophy.

# publications[].label
Natural History of Cardiomyopathy in Adult Dogs With Golden Retriever Muscular Dystrophy.

# publications[].label
A Blinded, Placebo-Controlled Systemic Gene Therapy Efficacy Study in the GRMD Model of Duchenne Muscular Dystrophy

# publications[].label
Use of the six-minute walk test to characterize golden retriever muscular dystrophy.

# publications[].label
Oxidative damage to urinary proteins from the GRMD dog and mdx mouse as biomarkers of dystropathology in Duchenne muscular dystrophy.

# publications[].label
Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials.

# publications[].label
Whole genome sequencing reveals a 7 base-pair deletion in DMD exon 42 in a dog with muscular dystrophy.

# publications[].label
Osteopontin is linked with AKT, FoxO1, and myostatin in skeletal muscle cells.

# publications[].label
OPN-a induces muscle inflammation by increasing recruitment and activation of pro-inflammatory macrophages.

# publications[].label
Sparing of the dystrophin-deficient cranial sartorius muscle is associated with classical and novel hypertrophy pathways in GRMD dogs.

# publications[].label
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.

# publications[].publisher
American Association for the Advancement of Science (AAAS)

# publications[].label
Infectious and inflammatory diseases of the CNS

# publications[].label
Gene therapies in canine models for Duchenne muscular dystrophy.

# publications[].label
Anabolic Characterization Of Immortalized Murine L6, And Canine Myok9 Skeletal Myotubes

# publications[].label
Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy.

# publications[].publisher
American Society for Clinical Investigation

# publications[].label
Clinical progression of X-linked muscular dystrophy in two German Shorthaired Pointers.

# publications[].publisher
American Veterinary Medical Association (AVMA)

# publications[].label
MCF7 Human Breast Cancer Cells Engineered to Stably Overexpress DEPTOR via CRISPR/Cas9 to Reduce Anabolic Capacity

# streetAddress
School of Veterinary Medicine and Biomedical Sciences; 4458 TAMU; 

# schools[]
School of Veterinary Medicine and Biomedical Sciences