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Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges

10 August 24 Articles ، Toshifumi Yokota Gene therapy for spinal muscular atrophy ، SMA gene-targeting treatments ، Antisense oligonucleotide therapy for SMA ، Onasemnogene abeparvovec gene replacement ، Risdiplam small molecule treatment ، Challenges in SMA therapies ، SMN1 and SMN2 gene therapy
Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges
This review discusses gene-targeting therapies for spinal muscular atrophy, covering antisense oligonucleotides, gene replacement, small molecules, and treatment challenges.
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Glucose Metabolism as a Pre-clinical Biomarker for the Golden Retriever Model of Duchenne Muscular Dystrophy

22 May 24 Articles Glucose metabolism in muscular dystrophy ، GRMD biomarker for Duchenne dystrophy ، PET/CT imaging in DMD research ، GLUT4 expression in dystrophic muscle ، Metabolic dysfunction in Duchenne muscular dystrophy ، Pre-clinical biomarkers for DMD ، Insulin response in muscular dystrophy models
Glucose Metabolism as a Pre-clinical Biomarker for the Golden Retriever Model of Duchenne Muscular Dystrophy
This study evaluates glucose metabolism as a biomarker in the GRMD model of Duchenne muscular dystrophy, analyzing GLUT4 expression, PET/CT imaging, and metabolic regulation.
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Impaired Glucose Tolerance in Adults with Duchenne and Becker Muscular Dystrophy

22 May 24 Articles DuchenneMuscularDystrophy ، BeckerMuscularDystrophy ، PatientCare ، DMDResearch ، MedicalResearch ، بیماری‌های_نادر ، GeneticDisorders ، MuscleResearch ، GlucoseTolerance ، MetabolicHealth ، BMDResearch ، ClinicalStudy ، DiabetesAwareness ، HealthScience ، ImpairedGlucoseTolerance ، Metabolism
Impaired Glucose Tolerance in Adults with Duchenne and Becker Muscular Dystrophy
  Abstract This study aimed to evaluate glucose tolerance in adult males with Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD) using an oral glucose tolerance test (OGTT). The study investigated whether body composition influences glucose response. Participants: 28 adults with dystrophinopathy (13 BMD, 15 DMD) and 12 non-dystrophic controls. Method: 75g glucose ingestion with fingertip blood sampling every 30 minutes for 2 hours. Results: Higher glucose levels in …
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Transcriptional dysregulation of autophagy in the muscle of a mouse model of Duchenne muscular dystrophy

19 May 24 Articles Autophagy dysregulation in DMD ، FoxO and TFEB in Duchenne dystrophy ، mTORC1 pathway and muscle degeneration ، Resveratrol therapy for muscular dystrophy ، Transcriptional regulation of autophagy ، SIRT1 activators and DMD treatment ، Phosphorylation-mediated suppression of FoxOs
Transcriptional dysregulation of autophagy in the muscle of a mouse model of Duchenne muscular dystrophy
This study examines transcriptional dysregulation of autophagy in Duchenne muscular dystrophy, highlighting FoxO and TFEB suppression and resveratrol's therapeutic role.
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Identifying hub genes and dysregulated pathways in Duchenne muscular dystrophy

19 May 24 Articles Hub genes in Duchenne dystrophy ، DMD dysregulated pathways ، Differentially expressed genes in DMD ، Protein-protein interaction network analysis ، Molecular mechanisms of DMD progression ، Gene expression studies in muscular dystrophy ، Therapeutic targets for Duchenne dystrophy
Identifying hub genes and dysregulated pathways in Duchenne muscular dystrophy
This research explores differentially expressed genes (DEGs) and hub genes in Duchenne muscular dystrophy, analyzing their role in disease progression and treatment targets.
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Essential Terminology for Every Reader of Dystrophy Articles

18 May 24 Useful information Muscular dystrophy terminology ، Scientific terms for dystrophy research ، Cohort studies in neuromuscular diseases ، Exome sequencing and genetics ، Understanding dystrophy research ، Basic terms in genetic studies ، Essential glossary for muscular dystrophy
Essential Terminology for Every Reader of Dystrophy Articles
This article provides essential terminology for understanding muscular dystrophy research, covering key scientific terms such as cohort studies and exome sequencing.
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Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy

17 May 24 Articles ، Akinori Nakamura Exon skipping therapy for DMD ، Dystrophin restoration in Duchenne dystrophy ، Antisense oligonucleotide therapy ، Gene therapy for muscular dystrophy ، PMOs and 2’OMeAO for exon skipping ، Multi-exon skipping strategies ، Clinical trials for Duchenne dystrophy treatments
Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy
This review explores exon skipping therapy for Duchenne muscular dystrophy, discussing preclinical and clinical trials, therapeutic challenges, and future developments.
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The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments

15 May 24 Articles ، Toshifumi Yokota Dysferlinopathies clinical features ، DYSF gene mutations and muscle weakness ، Muscle membrane repair disorders ، Genetic therapies for dysferlinopathies ، Pharmacological treatments for DYSF-related diseases ، Precision medicine in muscular dystrophies ، Animal models for dysferlinopathy research
The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments
This review examines dysferlinopathies, highlighting clinical features, molecular mechanisms, genetic therapies, and the latest research in treatment development.
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Duchenne muscular dystrophy: promising early-stage clinical trials to watch

21 March 24 Articles ، Toshifumi Yokota Duchenne muscular dystrophy clinical trials ، Early-stage DMD treatments ، Exon-skipping therapies for DMD ، Gene therapy for Duchenne dystrophy ، Micro-dystrophin gene therapy ، AON treatments for DMD ، New drugs for Duchenne muscular dystrophy
Duchenne muscular dystrophy: promising early-stage clinical trials to watch
This review highlights early-stage clinical trials for Duchenne muscular dystrophy, covering exon-skipping therapies, gene therapy, and novel treatment strategies.
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Wnt7a is Required for Regeneration of Dystrophic Skeletal Muscle

20 March 24 Articles ، Michael A. Rudnicki ، Ehsan Javandoost Wnt7a and muscle regeneration ، Duchenne muscular dystrophy therapy ، Muscle repair mechanisms in DMD ، Wnt7a injection for muscle healing ، Skeletal muscle recovery factors ، Stem cell activation in muscle repair ، Therapeutic potential of Wnt7a
Wnt7a is Required for Regeneration of Dystrophic Skeletal Muscle
This study examines the role of Wnt7a in muscle regeneration, highlighting its necessity for muscle repair and its potential as a therapy for Duchenne muscular dystrophy.
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Isolation of small extracellular vesicles from regenerating muscle tissue using Tangential Flow Filt

19 March 24 Articles ، Michael A. Rudnicki Extracellular vesicle isolation ، Muscle tissue EV purification ، Tangential Flow Filtration (TFF) for EVs ، Size Exclusion Chromatography (SEC) method ، Wnt7a and extracellular vesicles ، Muscle injury and EV release ، Bioactive vesicles in muscle regeneration
Isolation of small extracellular vesicles from regenerating muscle tissue using Tangential Flow Filt
This study presents an optimized protocol for isolating extracellular vesicles (EVs) from regenerating muscle tissue using TFF and SEC, ensuring purity and efficiency.
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Satellite Cell Heterogeneity in Skeletal Muscle Homeostasis

18 March 24 Articles ، Alessandra Sacco Satellite cell heterogeneity ، Skeletal muscle homeostasis ، Muscle stem cell differentiation ، Self-renewal in satellite cells ، Muscle regeneration mechanisms ، Stem cell fate in muscle repair ، Skeletal muscle stem cell diversity
Satellite Cell Heterogeneity in Skeletal Muscle Homeostasis
This article explores the role of satellite cell heterogeneity in skeletal muscle homeostasis, highlighting its impact on muscle repair, self-renewal, and differentiation.
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Several muscle diseases 5 : DYSF (Dysferlin) , Miyoshi Myopathy

20 February 24 Useful information ، muscle diseases DYSF gene mutation ، Dysferlin deficiency and muscle disease ، Miyoshi myopathy causes and symptoms ، Limb-girdle muscular dystrophy type 2B ، Progressive muscle weakness in dysferlinopathies ، Distal muscle atrophy disorders ، Therapies for Miyoshi myopathy
Several muscle diseases 5 : DYSF (Dysferlin) , Miyoshi Myopathy
This article explores DYSF mutations and Miyoshi Myopathy, detailing the role of dysferlin in muscle membrane repair and how its deficiency leads to muscle degeneration.
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Several muscle diseases 4 : Skeletal Muscle FSHD

19 February 24 Useful information ، muscle diseases Progressive muscle weakness disorders ، FSHD muscular dystrophy ، Facioscapulohumeral muscular dystrophy causes ، DUX4 gene expression in FSHD ، Skeletal muscle dystrophy symptoms ، Autosomal dominant muscle diseases ، FSHD genetic mechanisms
Several muscle diseases 4 : Skeletal Muscle FSHD
This article discusses facioscapulohumeral muscular dystrophy (FSHD), its genetic mechanisms, symptoms, and current research on potential treatments.
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