A5082424084- Muscle Physiology and Disorders
- Cardiomyopathy and Myosin Studies
- Congenital heart defects research
- Virus-based gene therapy research
- Tissue Engineering and Regenerative Medicine
- Muscle metabolism and nutrition
- Adipose Tissue and Metabolism
- Intracranial Aneurysms: Treatment and Complications
- Neurogenetic and Muscular Disorders Research
- Cerebral Palsy and Movement Disorders
- Cardiac Arrest and Resuscitation
- Cardiac Structural Anomalies and Repair
- Acute Ischemic Stroke Management
- Cardiovascular Function and Risk Factors
- Cardiovascular Disease and Adiposity
- Mechanical Circulatory Support Devices
- RNA Interference and Gene Delivery
- Vascular Malformations Diagnosis and Treatment
- Viral Infectious Diseases and Gene Expression in Insects
- Spinal Cord Injury Research
Texas A&M University
2016-2025
Wave Life Sciences (United States)
2018
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, membrane-stabilizing protein encoded DMD gene. Although mouse models provide insight into potential corrective therapy, data from genetically homologous large animals, such as dystrophin-deficient golden retriever (GRMD) model, may more readily translate to humans. To evaluate clinical translatability an adeno-associated virus serotype 9 vector (AAV9)–microdystrophin (μDys5)...
Duchenne muscular dystrophy (DMD) is an X-linked muscle disease with weakness, loss of ambulation, and premature death. DMD patients have reduced bone health, including decreased femur length (FL), density, fractures. The mdx mouse model has paradoxically greater FL, strength, positively correlating mass. Bone morphology not been extensively studied in the genetic homolog, golden retriever (GRMD). aim this study was to compare characteristics GRMD dogs understand their relationship function...
Abstract Endovascular embolization is a minimally‐invasive technique whereby blood vessels supplying pathological structures are selectively occluded with various embolic agents. In many scenarios, it desirable for the to distally penetrate level of microvasculature, which maximizes devascularization. Existing agents exhibit inconsistent distal penetration and have other limitations including tendency proximal reflux, patient pain during infusion, lack fluoroscopic radiopacity, potential...
Background Duchenne muscular dystrophy ( DMD ) is an X‐linked disease that causes progressive muscle weakness. Affected boys typically die from respiratory or cardiac failure. Golden retriever GRMD genetically homologous with and analogous skeletal disease. Previous studies have detailed features of cardiomyopathy in mostly young dogs. Cardiac not well characterized adult dogs, magnetic resonance CMR imaging been completed. Methods Results We evaluated echocardiography 24 dogs at different...
Metabolic dysfunction in Duchenne muscular dystrophy (DMD) is characterized by reduced glycolytic and oxidative enzymes, decreased abnormal mitochondria, ATP, increased stress. We analyzed glucose metabolism as a potential disease biomarker the genetically homologous golden retriever (GRMD) dog with molecular, biochemical, vivo imaging. Pelvic limb skeletal muscle left ventricle tissue from heart were mRNA profiling, qPCR, western blotting, immunofluorescence microscopy for primary...
We have examined the effects of intravenous (IV) delivery rAAVrh74.MHCK7. GALGT2 in golden retriever muscular dystrophy (GRMD) model Duchenne Muscular Dystrophy (DMD). After baseline testing, GRMD dogs were treated at 3 months age and reassessed 6 months. This 3–6 month range is a period rapid disease progression, thus offering relatively short window to establish treatment efficacy. Measures analyzed included muscle AAV transduction, transgene expression, -induced glycosylation, pathology,...
Background Duchenne muscular dystrophy (DMD) is an X-linked inherited myopathy that causes progressive skeletal and cardiac muscle disease. Heart lesions were described in the earliest DMD reports, cardiomyopathy now leading cause of death. However, diagnostics treatment for have lagged behind those appendicular respiratory Most animal model studies been done mdx mouse, which has a relatively mild form cardiomyopathy. Dogs with genetically homologous condition, Golden Retriever (GRMD),...
Abstract Background Duchenne muscular dystrophy (DMD) and genetically homologous golden retriever (GRMD) are X-linked conditions causing progressive muscle wasting cardiomyopathy. We previously defined a DMD-like dilated cardiomyopathy in adult GRMD dogs. The goal of this study was to extend our work characterize the early natural history cardiac stress response young Methods Age-matched (N=7), carrier (N=10), normal (N=8) littermates at 3, 6, 12 months age were prospectively enrolled....
Abstract Purpose For pediatric patients, extracorporeal membrane oxygenation (ECMO) remains the predominant mechanical circulatory support (MCS) modality for heart failure (HF) although survival to discharge rates remain between 50 and 60% these patients. The device-blood interface disruption of physiologic hemodynamics are significant contributors poor outcomes. Methods In this study, we evaluate preclinical feasibility a minimally invasive, non-blood-contacting DCC prototype temporary MCS....
Introduction: Duchenne muscular dystrophy (DMD) and the genetically homologous model, golden retriever (GRMD), are x-linked conditions that cause progressive muscle wasting cardiomyopathy. We previously defined a late onset DMD-like dilated cardiomyopathy in adult GRMD dogs aimed to extend work young used for preclinical studies. The goal of this study was characterize early natural history cardiac stress response Methods: A prospective imaging completed 10 8 age-matched normal littermates...