ArticleNature communications2017
Structure of human lysosomal acid α-glucosidase-a guide for the treatment of Pompe disease.
Article in Nature communications, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 121 papers.
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Who cites it
121 citing papers in PubMed, 295 citations in OpenAlex.
- Porcine serum maltase-glucoamylase: structure, kinetics, and inhibition.Journal of enzyme inhibition and medicinal chemistry · 2026Article
- AAV9-mediated GAA gene therapy following enzyme replacement therapy discontinuation in children with infantile-onset Pompe disease.EClinicalMedicine · 2026Article
- Eco-Friendly Synthesis, Physicochemical Characterization, and In Vitro Biological Evaluation of Plant-Derived Bioactive-Loaded Chitosan Nanoparticles Supported by Molecular Modeling Studies.Molecules (Basel, Switzerland) · 2026Article
- Differential Inhibitory Mechanisms of Myricetin and Dihydromyricetin on α-Glucosidase: A Combined Molecular Docking, Isothermal Titration Calorimetry and Surface Plasmon Resonance Study.Foods (Basel, Switzerland) · 2026Article
- Deoxynojirimycin derivatives as potent α-glucosidase inhibitors: in silico ADMET evaluation, molecular dynamics and in vitro validation studies.Molecular diversity · 2026Article
- Synthesis and evaluation of vanillin-based sulfonates as potent α-glucosidase inhibitors through in vitro, in vivo, and in silico approaches.Scientific reports · 2026Article
- Article
- Red Grape Pomace as a Valuable Source of Biomolecules With Inhibitory Effect on Pro-Inflammatory and Metabolic Syndrome-Associated Enzymes: Eco-Friendly Extraction and Mechanism of Inhibition.Food science & nutrition · 2026Article
- Unveiling the antidiabetic, anti-inflammatory and anti-hemolytic activities of Raphanus sativus leaves through in-vitro and in-silico studies.Scientific reports · 2026Article
- ADME/drug-likeness and functional properties of Punica granatum seeds supported with molecular docking, GC-MS and LC-MS/MS analysis.Scientific reports · 2026Article
- Combined In Vitro and Computational Investigations on Synthesized Sulfonamide-Based Antidiabetic Agents.Pharmaceuticals (Basel, Switzerland) · 2026Article
- Review
- In Situ Differential Analysis of α- and β-Glycosidase Activities in Lysosomes After Internalization Using Glucosylcerebroside-Based Liposomes.International journal of molecular sciences · 2026Article
- Evaluating Chronic Sex-Specific Changes in Glutamatergic Signaling Markers Following Traumatic Brain Injury.International journal of molecular sciences · 2026Article
- Longitudinal characterization of Gaac.1826dupA mice reveals the cardiac, myopathic and biochemical phenotypes of Pompe disease.Disease models & mechanisms · 2026Article
- Whole-Exome Sequencing Improves Risk Assessments of Adult Moyamoya Disease.Journal of clinical neurology (Seoul, Korea) · 2026Article
- Macroalgal Peptides with Predicted α-Glucosidase Inhibitory Activity: Preparation and Molecular Docking.Marine drugs · 2026Article
- Exploring the anti-diabetic potential of bis-Schiff bases of ibuprofen: insights into theRSC advances · 2026Article
- Machine learning-assisted optimization of ultrasound-ohmic processing of hawthorn vinegar: antidiabetic activity, phenolic profiling, and molecular docking insights.Frontiers in nutrition · 2026Article
- Article
61 more citing papers are in PubMed but not listed here.
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Authors and funding
9 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Pompe disease, a rare lysosomal storage disease caused by deficiency of the lysosomal acid α-glucosidase (GAA), is characterized by glycogen accumulation, triggering severe secondary cellular damage and resulting in progressive motor handicap and premature death. Numerous disease-causing mutations in the gaa gene have been reported, but the structural effects of the pathological variants were unknown. Here we present the high-resolution crystal structures of recombinant human GAA (rhGAA), the standard care of Pompe disease. These structures portray the unbound form of rhGAA and complexes thereof with active site-directed inhibitors, providing insight into substrate recognition and the molecular framework for the rationalization of the deleterious effects of disease-causing mutations. Furthermore, we report the structure of rhGAA in complex with the allosteric pharmacological chaperone N-acetylcysteine, which reveals the stabilizing function of this chaperone at the structural level.
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