ArticleCommunications biology2021
Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease.
Article in Communications biology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 1 of them a synthesis that pooled it.
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Who cites it
31 citing papers in PubMed, 1 synthesis or guideline pooled it, 33 citations in OpenAlex.
- What's new and what's next for gene therapy in Pompe disease?Expert opinion on biological therapy · 2022Pooled it
- Accumulation of membrane repair-associated proteins and mature myostatin are novel markers of muscle pathophysiology in Pompe disease.Acta neuropathologica communications · 2026Article
- RYR1Journal of translational medicine · 2026Article
- Pompe Disease: Pathogenesis, Molecular Mechanisms, Neurological Aspects, Diagnostics and Modern Therapeutic Approaches.International journal of molecular sciences · 2026Review
- Monitoring biological effects of somatic cell genome editing.Nature reviews. Genetics · 2026Review
- Engineering Functional Skeletal Muscle Tissues from Human Induced Pluripotent Stem Cells.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Engineered Muscle Tissue Analysis of Irisin Protection Against Glucocorticoid- and Cancer Cachexia-Induced Muscle Wasting and Cardiotoxicity.Biochip journal · 2026Article
- Biofabrication of a 3D human skeletal muscle microenvironment to study the early steps of fibrosis.Materials today. Bio · 2025Article
- Shaping 3D minimal model tissues with mechanical constraints to orchestrate muscle differentiation.Communications biology · 2025Article
- Review
- Leveraging microtopography to pattern multi-oriented muscle actuators.Biomaterials science · 2025Article
- Three-dimensional tissue engineered skeletal muscle modelling facioscapulohumeral muscular dystrophy.Brain : a journal of neurology · 2025Article
- Human Myobundle Platform for Studying the Role of Notch Signaling in Satellite Cell Phenotype and Function.Advanced healthcare materials · 2025Article
- Review
- Engineered Cardiac Tissues as a Platform for CRISPR-Based Mitogen Discovery.Advanced healthcare materials · 2025Article
- Engineering of tissue in microphysiological systems demonstrated by modelling skeletal muscle.Regenerative biomaterials · 2025Review
- A Micromanipulation-Actuated Large-Scale Screening to Identify Optimized Microphysiological Model Parameters in Skeletal Muscle Regeneration.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
- Engineered myovascular tissues for studies of endothelial/satellite cell interactions.Acta biomaterialia · 2024Article
- Endothelial-mesenchymal transition in skeletal muscle: Opportunities and challenges from 3D microphysiological systems.Bioengineering & translational medicine · 2024Review
- Bioengineered Model of Human LGMD2B Skeletal Muscle Reveals Roles of Intracellular Calcium Overload in Contractile and Metabolic Dysfunction in Dysferlinopathy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
Corrections and comments
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
Abstract
In Pompe disease, the deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA) causes skeletal and cardiac muscle weakness, respiratory failure, and premature death. While enzyme replacement therapy using recombinant human GAA (rhGAA) can significantly improve patient outcomes, detailed disease mechanisms and incomplete therapeutic effects require further studies. Here we report a three-dimensional primary human skeletal muscle ("myobundle") model of infantile-onset Pompe disease (IOPD) that recapitulates hallmark pathological features including reduced GAA enzyme activity, elevated glycogen content and lysosome abundance, and increased sensitivity of muscle contractile function to metabolic stress. In vitro treatment of IOPD myobundles with rhGAA or adeno-associated virus (AAV)-mediated hGAA expression yields increased GAA activity and robust glycogen clearance, but no improvements in stress-induced functional deficits. We also apply RNA sequencing analysis to the quadriceps of untreated and AAV-treated GAA
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.