ArticleProceedings of the National Academy of Sciences of the United States of America2008
Structural and mechanistic insight into the basis of mucopolysaccharidosis IIIB.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
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Who cites it
41 citing papers in PubMed, 89 citations in OpenAlex.
- Evaluation of GlcNAc-Configured Glycomimetics as Pharmacological Chaperones of NAGLU for the Treatment of Mucopolysaccharidosis IIIB.Biomolecules · 2026Article
- Expression and characterization of an endo-β-1,6-galactanase from Arabidopsis thaliana.The Biochemical journal · 2025Article
- Recent Progress of Small-molecule Inhibitors of O-GlcNAcase for Alzheimer's Disease.Mini reviews in medicinal chemistry · 2025Review
- Carbohydrate-active enzymes (CAZymes) in the gut microbiome.Nature reviews. Microbiology · 2022Review
- A GH89 human α-N-acetylglucosaminidase (hNAGLU) homologue from gut microbe Bacteroides thetaiotaomicron capable of hydrolyzing heparosan oligosaccharides.AMB Express · 2021Article
- Genotype to Phenotype: Identification of Mucopolysaccharidosis Type IIIB (Sanfilippo's B) Case Using Whole Exome Sequencing.Journal of pediatric genetics · 2021Article
- Temporospatial Development of Neuropathologic Findings in a Canine Model of Mucopolysaccharidosis IIIB.Veterinary pathology · 2021Article
- Trefoil factors share a lectin activity that defines their role in mucus.Nature communications · 2020Article
- Synthetic and Crystallographic Insight into Exploiting spChembiochem : a European journal of chemical biology · 2019Article
- A novel frameshift deletion inClinical case reports · 2018Article
- Utilizing ExAC to assess the hidden contribution of variants of unknown significance to Sanfilippo Type B incidence.PloS one · 2018Article
- High-Throughput Screen Fails to Identify Compounds That Enhance Residual Enzyme Activity of Mutant N-Acetyl-α-Glucosaminidase in Mucopolysaccharidosis Type IIIB.JIMD reports · 2018Article
- Update of the spectrum of mucopolysaccharidoses type III in Tunisia: identification of three novel mutations and in silico structural analysis of the missense mutations.World journal of pediatrics : WJP · 2017Article
- Dissecting PUGNAc-mediated inhibition of the pro-survival action of insulin.Glycobiology · 2016Article
- The structure of a glycoside hydrolase 29 family member from a rumen bacterium reveals unique, dual carbohydrate-binding domains.Acta crystallographica. Section F, Structural biology communications · 2016Article
- Azasugar inhibitors as pharmacological chaperones for Krabbe disease.Chemical science · 2015Article
- Using support vector machines to improve elemental ion identification in macromolecular crystal structures.Acta crystallographica. Section D, Biological crystallography · 2015Article
- Mucin glycan foraging in the human gut microbiome.Frontiers in genetics · 2015Review
- "Genotype-first" approaches on a curious case of idiopathic progressive cognitive decline.BMC medical genomics · 2014Article
- Unravelling the multiple functions of the architecturally intricate Streptococcus pneumoniae β-galactosidase, BgaA.PLoS pathogens · 2014Article
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mucopolysaccharidosis III (MPS III) has four forms (A-D) that result from buildup of an improperly degraded glycosaminoglycan in lysosomes. MPS IIIB is attributable to the decreased activity of a lysosomal alpha-N-acetylglucosaminidase (NAGLU). Here, we describe the structure, catalytic mechanism, and inhibition of CpGH89 from Clostridium perfringens, a close bacterial homolog of NAGLU. The structure enables the generation of a homology model of NAGLU, an enzyme that has resisted structural studies despite having been studied for >20 years. This model reveals which mutations giving rise to MPS IIIB map to the active site and which map to regions distant from the active site. The identification of potent inhibitors of CpGH89 and the structures of these inhibitors in complex with the enzyme suggest small-molecule candidates for use as chemical chaperones. These studies therefore illuminate the genetic basis of MPS IIIB, provide a clear biochemical rationale for the necessary sequential action of heparan-degrading enzymes, and open the door to the design and optimization of chemical chaperones for treating MPS IIIB.
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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.