Guy L. Odom
YOU?
Author Swipe
View article: Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle Open
Background: Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy without an effective treatment, caused by mutations in the DMD gene, leading to the absence of dystrophin. DMD results in muscle weakness, loss of ambulat…
View article: WITHDRAWN: Cell based dATP delivery as a therapy for chronic heart failure
WITHDRAWN: Cell based dATP delivery as a therapy for chronic heart failure Open
Withdrawal Statement The authors have withdrawn this manuscript because the senior authors have identified inconsistencies in the data presented in this manuscript, which affect the reliability of the main conclusions. In keeping with our …
View article: Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model Open
Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are currently in clinical trials, but have not been thoroughly investigated for their efficacy on cardiomyopathy progression to heart failure. We previously …
View article: Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model
Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model Open
Gene replacement for Duchenne muscular dystrophy (DMD) with micro-dystrophins has entered clinical trials, but efficacy in preventing heart failure is unknown. Although most patients with DMD die from heart failure, cardiomyopathy is undet…
View article: Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects
Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects Open
Gene therapy approaches for DMD using recombinant adeno-associated viral (rAAV) vectors to deliver miniaturized (or micro) dystrophin genes to striated muscles have shown significant progress. However, concerns remain about the potential f…
View article: Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate
Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate Open
Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. Administration of recombinant adeno-associa…
View article: Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16–17
Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16–17 Open
Mutation of the gene encoding dystrophin leads to Duchenne and Becker muscular dystrophy (DMD and BMD). Currently, dystrophin is thought to function primarily as a structural protein, connecting the muscle cell actin cytoskeleton to the ex…
View article: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy Open
Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9. He…
View article: Translation of Cardiac Myosin Activation With 2-Deoxy-ATP to Treat Heart Failure Via an Experimental Ribonucleotide Reductase-Based Gene Therapy
Translation of Cardiac Myosin Activation With 2-Deoxy-ATP to Treat Heart Failure Via an Experimental Ribonucleotide Reductase-Based Gene Therapy Open
Despite recent advances, chronic heart failure remains a significant and growing unmet medical need, reaching epidemic proportions carrying substantial morbidity, mortality, and costs. A safe and convenient therapeutic agent that produces …
View article: Validation of ultrasonography for non‐invasive assessment of diaphragm function in muscular dystrophy
Validation of ultrasonography for non‐invasive assessment of diaphragm function in muscular dystrophy Open
Key points Duchenne muscular dystrophy (DMD) is a severe, degenerative muscle disease that is commonly studied using the mdx mouse. The mdx diaphragm muscle closely mimics the pathophysiological changes in DMD muscles. mdx diaphragm force …
View article: 501. In Vivo Gene Editing for Duchenne Muscular Dystrophy
501. In Vivo Gene Editing for Duchenne Muscular Dystrophy Open
Gene replacement therapies utilizing adeno-associated viral vector delivery of truncated versions of dystrophin (mini- and microdystrophins) hold great potential for the treatment of Duchenne muscular dystrophy (DMD). However, these trunca…
View article: 380. Therapeutic Capacity of AAV-Delivered Muscle-Specific-Expressed Micro-Utrophin (ΔR4-R21/ΔCT) in mdX4cv Skeletal Muscles
380. Therapeutic Capacity of AAV-Delivered Muscle-Specific-Expressed Micro-Utrophin (ΔR4-R21/ΔCT) in mdX4cv Skeletal Muscles Open
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder caused by dystrophin mutations. Utrophin is a dystrophin paralogue that can prevent necrosis in the mdx mouse DMD model. We designed an expression plasmid containing a m…
View article: Progress and prospects of gene therapy clinical trials for the muscular dystrophies
Progress and prospects of gene therapy clinical trials for the muscular dystrophies Open
Clinical trials represent a critical avenue for new treatment development, where early phases (I, I/II) are designed to test safety and effectiveness of new therapeutics or diagnostic indicators. A number of recent advances have spurred re…
View article: 11. Gene Therapy Mediated Increase in dATP Improves Cardiac Performance in Models of Systolic Heart Failure
11. Gene Therapy Mediated Increase in dATP Improves Cardiac Performance in Models of Systolic Heart Failure Open
Heart Failure results in more deaths and hospitalizations than almost any other single cause. We are exploring gene therapy treatment strategies that increase 2 deoxy-adenosine triphosphate (dATP) levels via cardiac specific expression of …
View article: 611. Alpha-Dystrobrevin-3 Prevents Myopathy and Restores Diaphragm Function in the Alpha-Dystrobrevin Null Mouse
611. Alpha-Dystrobrevin-3 Prevents Myopathy and Restores Diaphragm Function in the Alpha-Dystrobrevin Null Mouse Open
The dystrophin-glycoprotein complex (DGC) primarily functions as a structural element enabling a linkage between the cytoskeleton and the extracellular matrix. Alpha-dystrobrevin (α-Db), a dystrophin subfamily member, is a component of the…
View article: Recombinant adeno‐associated viral (<scp>rAAV</scp>) vectors mediate efficient gene transduction in cultured neonatal and adult microglia
Recombinant adeno‐associated viral (<span>rAAV</span>) vectors mediate efficient gene transduction in cultured neonatal and adult microglia Open
Microglia are a specialized population of myeloid cells that mediate CNS innate immune responses. Efforts to identify the cellular and molecular mechanisms that regulate microglia behaviors have been hampered by the lack of effective tools…