Explanipedia

EXTH-59. Supporting development of novel brain tumour therapies through global collaboration: early insights from a novel therapeutics accelerator Open

Charlotte Aitken, Katie Bushby, Camille Goetz, Nicky Huskens, Karen Noble , et al. · 2025

Researchers developing novel therapies for brain tumours must navigate numerous challenges, including selection of suitable preclinical studies, safety, trial design, evolving regulatory landscapes, and securing sustained funding. To addre…

AN INTERNATIONAL NOVEL THERAPEUTICS ACCELERATOR FOR BRAIN TUMOURS: EARLY IMPACT AND LESSONS LEARNED Open

Charlotte Aitken, Katie Bushby, Camille Goetz, Petra Hamerlik, Nicky Huskens , et al. · 2025

AIMS Developing a translational programme for brain tumours involves navigating numerous challenges, including robustness of preclinical studies, safety, clinical trial design, understanding and addressing regulatory require- ments, and fu…

Corrigendum: Enhancing AAV-microdystrophin gene therapy after repeat dosing by blocking phagocytosis Open

Rita Spathis, Deeva Robles Kuriplach, Sabrina Narvesen, Matthew Eybs, Karen Huang , et al. · 2025

[This corrects the article DOI: 10.3389/fimmu.2025.1527840.].

The effect of IL-1β inhibitor canakinumab (Ilaris®) on IL-6 production in human skeletal muscle cells Open

Anna Cordeiro, Fiorella Morales, Maria del Carmen Parquet, Kitipong Uaesoontrachoon, Joyce Rowsell , et al. · 2025

Muscle inflammation is one of the hallmarks of Duchenne muscular dystrophy (DMD). Dystrophin-deficient skeletal muscle cells produce higher levels of pro-inflammatory cytokines such as interleukin 1β (IL-1β) in response to toll-like recept…

Enhancing AAV-microdystrophin gene therapy after repeat dosing by blocking phagocytosis Open

Rita Spathis, Deeva Robles Kuriplach, Sabrina Narvesen, Matthew Eybs, Karen Huang , et al. · 2025

Background Inefficient transduction is a major limitation in achieving therapeutic levels of AAV-delivered microdystrophin capable of improving muscle function in patients with Duchenne muscular dystrophy. Additionally, some patients exper…

High mobility group box 1 (HMGB1) is a potential disease biomarker in cell and mouse models of Duchenne muscular dystrophy Open

Rebecca A. Slick, J. R. Sutton, Margaret Haberman, Benjamin S. O’Brien, Jennifer A Tinklenberg , et al. · 2024

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder affecting 1:3500 male births and is associated with myofiber degeneration, regeneration, and inflammation. Glucocorticoid treatments have been the standard of care …

OTHR-06. A NEW INTERNATIONAL, MULTIDISCIPLINARY ACCELERATOR PROGRAMME FOR NOVEL BRAIN TUMOUR THERAPIES Open

Charlotte Aitken, Nicky Huskens, Katie Bushby, Karen Noble, Petra Hamerlik , et al. · 2024

BACKGROUND Developing a comprehensive translational programme for brain tumours involves navigating numerous challenges, including robustness of preclinical studies, safety, clinical trial design, regulatory issues, and commercialisation. …

A population-based study of children suggests blunted morning cortisol rhythms are associated with alterations of the systemic inflammatory state Open

Runia Roy, Utkarsh J. Dang, Kim M. Huffman, Tchilabalo Dilezitoko Alayi, Yetrib Hathout , et al. · 2023

We provide preliminary data on diurnal fluctuations of inflammatory cytokines in saliva in a population-based cohort of children. Correlation of morning and evening cortisol levels with inflammatory cytokines in the same saliva samples sho…

Loss of calpain3b in Zebrafish, a Model of Limb-Girdle Muscular Dystrophy, Increases Susceptibility to Muscle Defects Due to Elevated Muscle Activity Open

Sergey V. Prykhozhij, Lucía Cáceres, Kevin Ban, Anna Cordeiro, Kanneboyina Nagaraju , et al. · 2023

Limb-Girdle Muscular Dystrophy Type R1 (LGMDR1; formerly LGMD2A), characterized by progressive hip and shoulder muscle weakness, is caused by mutations in CAPN3. In zebrafish, capn3b mediates Def-dependent degradation of p53 in the liver a…

Death and Disability Reported with Cases of Vaccine Anaphylaxis Stratified by Administration Setting: An Analysis of the Vaccine Adverse Event Reporting System from 2017 to 2022 Open

Rachel C. Klosko, Sarah Lynch, Danielle L. Cabral, Kanneboyina Nagaraju, Yvonne Johnston , et al. · 2023

The serious nature of post-vaccination anaphylaxis requires healthcare professionals to be adequately trained to respond to these hypersensitivity emergencies. The aim of this study was to compare outcomes reported with cases of vaccine an…

Urine proteomics by mass spectrometry identifies proteins involved in key pathogenic pathways in patients with juvenile dermatomyositis Open

Melissa Morales, Tchilabalo Dilezitoko Alayi, Shefa M. Tawalbeh, Agnes V. Sydenstricker, Rita Spathis , et al. · 2023

Objectives To identify and validate biomarkers in JDM patients using a multiplexing tandem mass tag urine proteome profiling approach. Methods First morning void urine samples were collected from JDM patients (n = 20) and healthy control s…

Comparative study of certain Biochemistry parameters of serum constituents with reference to age Open

Amala Arockia Raj Fr. T. S.J., Kanneboyina Nagaraju, Muniya Naik M, A Jayasankar, P Ramanjaneyulu , et al. · 2023

The objective of the current study was to explore the various serum biochemical parameters in people with different ages.When compared to the young age group, biochemical investigation of some instances showed a substantial increase in blo…

Mechanism of action and therapeutic route for a muscular dystrophy caused by a genetic defect in lipid metabolism Open

Mahtab Tavasoli, Sarah Lahire, Stanislav Sokolenko, Robyn J. Novorolsky, Sarah Anne Reid , et al. · 2022

A mouse model of inherited choline kinase β-deficiency presents with specific cardiac abnormalities and a predisposition to arrhythmia Open

Mahtab Tavasoli, Tiam Feridooni, Hirad Feridooni, Stanislav Sokolenko, Abhishek Mishra , et al. · 2022

The CHKB gene encodes choline kinase β, which catalyzes the first step in the biosynthetic pathway for the major phospholipid phosphatidylcholine. Homozygous loss-of-function variants in human CHKB are associated with a congenital muscular…

Secreted acid sphingomyelinase as a potential gene therapy for limb girdle muscular dystrophy 2B Open

Daniel C. Bittel, Sen Chandra Sreetama, Goutam Chandra, Robin J. Ziegler, Kanneboyina Nagaraju , et al. · 2022

Efficient sarcolemmal repair is required for muscle cell survival, with deficits in this process leading to muscle degeneration. Lack of the sarcolemmal protein dysferlin impairs sarcolemmal repair by reducing secretion of the enzyme acid …

Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy Open

James S. Novak, Rita Spathis, Utkarsh J. Dang, Alyson A. Fiorillo, Ravi Hindupur , et al. · 2021

Recently, the Food and Drug Administration granted accelerated approvals for four exon skipping therapies –Eteplirsen, Golodirsen, Viltolarsen, and Casimersen –for Duchenne Muscular Dystrophy (DMD). However, these treatments have only demo…

Validation of Chemokine Biomarkers in Duchenne Muscular Dystrophy Open

Michael Ogundele, Jesslyn S. Zhang, Mansi V. Goswami, Marissa Barbieri, Utkarsh J. Dang , et al. · 2021

Duchenne muscular dystrophy (DMD) is a progressive muscle disease involving complex skeletal muscle pathogenesis. The pathogenesis is triggered by sarcolemma instability due to the lack of dystrophin protein expression, leading to Ca2+ inf…

Biomarker-focused multi-drug combination therapy and repurposing trial in mdx mice Open

Michael Ziemba, Molly Barkhouse, Kitipong Uaesoontrachoon, Mamta Giri, Yetrib Hathout , et al. · 2021

Duchenne muscular dystrophy is initiated by dystrophin deficiency, but downstream pathophysiological pathways such as membrane instability, NFĸB activation, mitochondrial dysfunction, and induction of TGFβ fibrosis pathways are thought to …

Mitochondrial dysfunction and consequences in calpain-3-deficient muscle Open

Vanessa E. Jahnke, Jennifer M. Peterson, Jan van der Meulen, J Boehler, Kitipong Uaesoontrachoon , et al. · 2020

One-year Treatment of Morpholino Antisense Oligomer Improves Skeletal and Cardiac Muscle Functions in Dystrophic mdx Mice Open

Bo Wu, Bin Xiao, Caryn Cloer, Mona Shaban, Arpana Sali , et al. · 2020

Antisense therapy has been successful to skip targeted dystrophin exon with correction of frameshift and nonsense mutations of Duchenne muscular dystrophy (DMD). Systemic production of truncated but functional dystrophin proteins has been …

Full programme Open

Max Damian, Annamaria Deluca, Kanneboyina Nagaraju, Dominic J. Wells, Jennifer E. Morgan , et al. · 2020

Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment Open

Xu Liu, Yashuo Wang, Jennifer S. Gutierrez, Jesse M. Damsker, Kanneboyina Nagaraju , et al. · 2020

Significance Corticosteroids that are currently in clinical use bind to the glucocorticoid receptor (GR) to exert antiinflammation effects, yet are associated with undesirable side effects. Vamorolone is a recently developed drug for Duche…

Muscle Weakness in Myositis: MicroRNA‐Mediated Dystrophin Reduction in a Myositis Mouse Model and Human Muscle Biopsies Open

Travis B. Kinder, Christopher R. Heier, Christopher B. Tully, Jack H. Van der Muelen, Eric P. Hoffman , et al. · 2020

Objective Muscle inflammation is a feature in myositis and Duchenne muscular dystrophy ( DMD ). Autoimmune mechanisms are thought to contribute to muscle weakness in patients with myositis. However, a lack of correlation between the extent…

Additional file 2 of Mitochondrial dysfunction and consequences in calpain-3-deficient muscle Open

Vanessa E. Jahnke, Jennifer M. Peterson, Jan van der Meulen, J Boehler, Kitipong Uaesoontrachoon , et al. · 2020

Additional file 2: FFold changes in WT and calpain-3 deficient myoblasts and myotubes.

A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment Open

Daniela Gois Beghini, Samuel Iwao Maia Horita, Liana Monteiro da Fonseca Cardoso, Luiz Anastácio Alves, Kanneboyina Nagaraju , et al. · 2019

Muscular dystrophies (MD) are a group of genetic diseases that lead to skeletal muscle wasting and may affect many organs (multisystem). Unfortunately, no curative therapies are available at present for MD patients, and current treatments …

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function Open

Eric P. Hoffman, Benjamin D. Schwartz, L. Mengle-Gaw, Edward C. Smith, Diana Castro , et al. · 2019

This study provides Class IV evidence that for boys with DMD, vamorolone demonstrated possible efficacy compared to a natural history cohort of glucocorticoid-naive patients and appeared to be tolerated.

Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset Open

Jesse M. Damsker, Michaelyn R. Cornish, Priya Kanneboyina, Ila Kanneboyina, Qìng Yu , et al. · 2019

Orthogonal Analysis of Dystrophin Protein and Mrna As A Surrogate Outcome for Drug Development Open

Kitipong Uaesoontrachoon, Sadish Srinivassane, Jordan Warford, Kevork Mekhssian, Hélène Montpetit , et al. · 2019

Aim: Detection of drug-induced dystrophin in patient muscle biopsy is a surrogate outcome measure for Duchenne muscular dystrophy. We sought to establish and validate an orthogonal approach to measurement of dystrophin protein and R…

AMP‐activated protein kinase signaling regulated expression of urea cycle enzymes in response to changes in dietary protein intake Open

Sandra Kirsch Heibel, Peter J. McGuire, Nantaporn Haskins, Himani D. Majumdar, Sree Rayavarapu , et al. · 2019

Abundance of urea cycle enzymes in the liver is regulated by dietary protein intake. Although urea cycle enzyme levels rise in response to a high‐protein (HP) diet, signaling networks that sense dietary protein intake and trigger changes i…

Fibroadipogenic progenitors are responsible for muscle loss in limb girdle muscular dystrophy 2B Open

Marshall W. Hogarth, Aurélia Defour, Christopher A. Lazarski, Eduard Gallardo, Jordi Díaz‐Manera , et al. · 2019

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