Explanipedia

Characterization of Gait Kinematics and Muscle Function in Becker Muscular Dystrophy Pigs: a pilot study Open

Aaron B. Morton, Jacob Kendra, Shadi Golpasandi, M.L. Mackey, Haley Russell , et al. · 2025

Vertebrate animal models of Becker muscular dystrophy (BMD) have been developed. Here, we characterized the gait kinematics and muscle function of a naturally occurring BMD pig model of dystrophin insufficiency. BMD pigs tended to have alt…

Lymphatic dysfunction is linked to disease pathogenesis in Duchenne muscular dystrophy animal models Open

Balanand Subramanian, Steven R. Johnson, A. Narayanan, Wei Wang, Bonnie L. Seaberg , et al. · 2025

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder characterized by progressive muscle weakness and inflammation caused by mutations in the DMD gene. Chronic inflammation in DMD exacerbates the complications associated w…

Beta-hydroxy-beta-methylbutyrate (HMB) improves daily activity and whole-body protein metabolism in Duchenne muscular dystrophy dogs: a pilot study Open

Peter P. Nghiem, Alexis Rutledge, Kyle Tehas, Corine Kaderli, Mark Poling , et al. · 2025

MCF7 breast cancer anabolic capacity reduced with CRISPR/Cas9-mediated stable overexpression of DEPTOR Open

J. William Deaver, Patrick J. Ryan, Colleen L. O’Reilly, Selina Uranga, Sara Mata López , et al. · 2025

We identify DEPTOR as a predictor of mortality in breast cancer and show that precision gene editing to restore DEPTOR expression in breast cancer slows cell growth by inhibiting mTOR activity.

Bone measurements interact with phenotypic measures in canine Duchenne muscular dystrophy Open

Sarah M. Schneider, M.L. Mackey, Shane R. Wilkinson, Lee‐Jae Guo, Peter P. Nghiem · 2025

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, and fractures. The mdx mouse mod…

Early Natural History of Cardiomyopathy and Cardiac Stress Response in Young Dogs with Golden Retriever Muscular Dystrophy Open

Lee‐Jae Guo, Peter P. Nghiem, Amanda K. Bettis, Jonathan H. Soslow, Christopher F. Spurney , et al. · 2024

Background Duchenne muscular dystrophy (DMD) and genetically homologous golden retriever muscular dystrophy (GRMD) are X-linked conditions causing progressive muscle wasting and cardiomyopathy. We previously defined a DMD-like dilated card…

Non-essential Amino Acids Are Needed to Attenuate ICU Acquired Muscle Weakness (ICU-AW) in a Pig Model of Sepsis Recovery Open

Nicolaas E.P. Deutz, Gabriella A Ten Have, Peter P. Nghiem, M.L. Mackey, Sarah E. Rice , et al. · 2024

Compromised muscle function and physical activity during recovery of sepsis in an ICU-acquired weakness translational pig model Open

G. A. Ten Have, Peter P. Nghiem, M.L. Mackey, Caitlin Sas, S.A. Rice , et al. · 2023

Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy Open

Alan J. Russell, Mike Duvall, Ben Barthel, Ying Qian, Angela K. Peter , et al. · 2023

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by absence of the protein dystrophin, which acts as a structural link between the basal lamina and contractile machinery to stabilize muscle membranes in response to mecha…

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy Open

Sharla M. Birch, Michael W. Lawlor, Thomas J. Conlon, Lee‐Jae Guo, Julie M. Crudele , et al. · 2023

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene. Although mouse models of DMD provide insight into the potential of a cor…

(-)-Agelastatin A is a Potent Modulator of Osteopontin Expression in Normal and Dystrophic Canine Myoblasts Open

Madison Feng, Sara Mata López, Peter P. Nghiem · 2023

(-)-Agelastatin A [(-)-AA] is a naturally occurring alkaloid of marine sponge origin that is a potent modulator of osteopontin. The latter is a multifunctional cytokine expressed in many types of cells and is involved with several importan…

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 Open

Paul T. Martin, Deborah A. Zygmunt, Anna Ashbrook, Sonia Hamilton, Davin Packer , et al. · 2021

We have examined the effects of intravenous (IV) delivery of rAAVrh74.MHCK7. GALGT2 in the golden retriever muscular dystrophy (GRMD) model of Duchenne Muscular Dystrophy (DMD). After baseline testing, GRMD dogs were treated at 3 months of…

Osteopontin (OPN) Downregulatory Alkaloid (-)-Agelastatin A Prevents Muscle Damage in a Mouse Model of Duchenne Muscular Dystrophy Open

Madison Feng, Sara Mata López, Soraya Manaviazar, Hamish A. Watson, Karl J. Hale , et al. · 2021

(-)-Agelastatin A (AA) in 1,2-propanediol (3-deoxy-DL-glycerol) elicits a dose-dependent decrease in OPN mRNA expression in canine Duchenne Muscular Dystrophy (DMD) myoblasts at doses ranging from 0.01 nM-30 nM. When intraperitoneally admi…

Osteopontin (OPN) Downregulatory Alkaloid (-)-Agelastatin A Prevents Muscle Damage in a Mouse Model of Duchenne Muscular Dystrophy Open

Madison Feng, Sara Mata López, Soraya Manaviazar, Hamish A. Watson, Karl J. Hale , et al. · 2021

(-)-Agelastatin A (AA) in 1,2-propanediol (3-deoxy-DL-glycerol) elicits a dose-dependent decrease in OPN mRNA expression in canine Duchenne Muscular Dystrophy (DMD) myoblasts at doses ranging from 0.01 nM-30 nM. When intraperitoneally admi…

Regulation of cellular anabolism by mTOR: or how I learned to stop worrying and love translation Open

J. William Deaver, Sara Mata López, Patrick J. Ryan, Peter P. Nghiem, Steven E. Riechman , et al. · 2020

Sparing of the Dystrophin-Deficient Cranial Sartorius Muscle Is Associated with Classical and Novel Hypertrophy Pathways in GRMD Dogs Open

Peter P. Nghiem, Eric P. Hoffman, Priya Mittal, Kristy J. Brown, Scott J. Schatzberg , et al. · 2020

Both Duchenne and golden retriever muscular dystrophy (GRMD) are caused by dystrophin deficiency. The Duchenne muscular dystrophy sartorius muscle and orthologous GRMD cranial sartorius (CS) are relatively spared/hypertrophied. We complete…

Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies Open

Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan, Martin K. Childers, Juan Li , et al. · 2020

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder in which the loss of dystrophin causes progressive degeneration of skeletal and cardiac muscle. Potential therapies that carry substantial risk, such as gene and cell-base…

Correction: Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy Open

Sara Mata López, Cynthia Balog-Alvarez, Stanislav Vitha, Amanda K. Bettis, Emily H. Canessa , et al. · 2020

[This corrects the article DOI: 10.1371/journal.pone.0228072.].

Oxidative damage to urinary proteins from the GRMD dog and mdx mouse as biomarkers of dystropathology in Duchenne muscular dystrophy Open

Jessica R. Terrill, Basma A. Al-Mshhdani, Marisa N. Duong, Catherine Wingate, Zahra Abbas , et al. · 2020

Duchenne muscular dystrophy (DMD) is a lethal, X-chromosome linked muscle-wasting disease affecting about 1 in 3500-6000 boys worldwide. Myofibre necrosis and subsequent loss of muscle mass are due to several molecular sequelae, such as in…

Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy Open

Sara Mata López, Cynthia Balog-Alvarez, Stanislav Vitha, Amanda K. Bettis, Emily H. Canessa , et al. · 2020

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish the expression of dystrophin protein. Dogs with the genetic homologue, golden retriever muscular dystrophy dog (GRMD), have a splice site mutation that l…

Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models Open

Yafeng Song, Leon Morales, Alock Malik, Andrew Frederick Mead, Christopher Greer , et al. · 2019

Natural History of Cardiomyopathy in Adult Dogs With Golden Retriever Muscular Dystrophy Open

Lee‐Jae Guo, Jonathan H. Soslow, Amanda K. Bettis, Peter P. Nghiem, Kevin J. Cummings , et al. · 2019

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 muscular dystrophy ( GRMD ) is genetically hom…

Correction to: Gene therapies in canine models for Duchenne muscular dystrophy Open

Peter P. Nghiem, Joe N. Kornegay · 2019

A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20 Open

Sara Mata López, James J. Hammond, Madison B. Rigsby, Cynthia Balog-Alvarez, Joe N. Kornegay , et al. · 2018

This spontaneous canine dystrophinopathy occurred due to a novel mutation in the minor DMD mutation hotspot (between exons 2 through 20). Perpetuating this line could allow for preclinical testing of genetic therapies targeted to this area…

Glucose Metabolism as a Pre-clinical Biomarker for the Golden Retriever Model of Duchenne Muscular Dystrophy Open

Sarah M. Schneider, Vidya Sridhar, Amanda K. Bettis, Heather Heath-Barnett, Cynthia Balog-Alvarez , et al. · 2018

Altered glucose metabolism in skeletal and cardiac muscle of GRMD dogs can be monitored with molecular, biochemical, and in vivo imaging studies and potentially utilized as a biomarker for disease progression and therapeutic response.

Whole genome sequencing reveals a 7 base-pair deletion in DMD exon 42 in a dog with muscular dystrophy Open

Peter P. Nghiem, Luca Bello, Cindy Balog‐Alvarez, Sara Mata López, Amanda K. Bettis , et al. · 2016

OPN‐a induces muscle inflammation by increasing recruitment and activation of pro‐inflammatory macrophages Open

Gina M. Many, Yasuyuki Yokosaki, Kitipong Uaesoontrachoon, Peter P. Nghiem, Luca Bello , et al. · 2016

New Findings What is the central question of this study? What is the functional relevance of OPN isoform expression in muscle pathology? What is the main finding and its importance? The full‐length human OPN‐a isoform is the most pro‐infla…

Peter P. Nghiem YOU? Author Swipe