bims-meglyc Biomed News
on Metabolic disorders affecting glycosylation
Issue of 2025–06–29
four papers selected by
Silvia Radenkovic, UMC Utrecht



  1. Cell Mol Life Sci. 2025 Jun 27. 82(1): 257
      SLC35A2-CDG is an X-linked congenital disorder of glycosylation (CDG), characterized by defective UDP-galactose transport into the Golgi and endoplasmic reticulum and consequent insufficient galactosylation of glycans. Clinically, this translates into a range of predominantly neurological symptoms. Although the pathomechanism of this disorder is not fully understood, oral galactose supplementation has led to clinical and biochemical improvement in some patients. Here, we show that protein glycosylation (N- and O-linked) was only minimally disturbed in SLC35A2-CDG patient-derived fibroblasts. However, lipid glycosylation was significantly impaired, with accumulation of glucosylceramide and deficiency of digalactosylated glycosphingolipids (GSLs) and complex gangliosides. Galactose supplementation increased UDP-galactose, its transport into the Golgi, and improved deficient GSL synthesis through direct incorporation of the provided galactose. This improved GSL homeostasis in all patient-derived fibroblasts and in another SLC35A2 deficient cell model (CHO-Lec8). Additionally, SLC35A2-CDG serum analysis identified hydroxylated GSLs, particularly GM3, as potential disease biomarkers. Given the essential role of gangliosides in central nervous system function, their deficiency is likely a key factor in the neurological involvement of this disorder. These findings pave the way for new nutritional therapies with GSL supplements and highlight the importance of studying lipid glycosylation to better understand the complex pathophysiology of CDG.
    Keywords:  CDG; GSL; Gangliosides; Glycosphingolipids; SLC35A2-CDG; Tracer metabolomics
    DOI:  https://doi.org/10.1007/s00018-025-05759-w
  2. Mol Genet Metab. 2025 Jun 15. pii: S1096-7192(25)00169-6. [Epub ahead of print]145(4): 109178
       OBJECTIVE: The most common congenital disorders of glycosylation (CDG) is the phosphomannomutase 2 (PMM2) deficiency (PMM2-CDG). PMM2-CDG is a complex genetic disorder that often found in infancy or early childhood with a clinically heterogeneous variety of neurological and non- neurological symptoms. To expand the phenotypic and genetic spectrum of PMM2-CDG, we summarized the characteristics of 20 Chinese patients.
    METHODS: All patients were diagnosed by genetic analysis. Clinical characteristics, genotypes, imaging, electrophysiological, and metabolic data were analyzed retrospectively.
    RESULTS: Twelve males and eight females with PMM2-CDG were included. The median age at diagnosis was 12.0 (ranging from 6.0 to 52.0) months, while the median age at final follow-up was 10.3 (ranging from 5.1 to 12.8) years. All patients exhibited multisystem symptoms and various neurological symptoms were observed. Developmental delay was the primary initial symptoms. Dystaxia, growth retardation and liver damage were also common phenotypes. Cerebellar atrophy was the characteristic abnormality on brain imaging. Fourteen variants of the PMM2 gene were identified, of which five, c.82A > G (p.M28V), c.551C > T (p. P184L), c.640G > T (p.G214C), c.656A > T (p.E219V) and c.712C > G (p.R238G), were newly reported. The most prevalent variant was c.430 T > C (p.F144L), which was identified in 65.0 % of patients, followed by c.395 T > C (p.I132T). Consistent with reports from other populations, missense variants constituted the predominant type of PMM2 gene alterations.
    CONCLUSION: PMM2-CDG presents as a multi-system disease with diverse clinical phenotypes, posing challenges to early identification and diagnosis. The most common pathogenic variant in this Chinese cohort was c.430 T > C (p.F144L), which is close to, but different from, the common pathogenic variant c.422G > A (p.R141H) among European PMM2-CDG patients.
    Keywords:  Chinese children; Congenital disorders of glycosylation; PMM2 variants; Phosphomannomutase 2
    DOI:  https://doi.org/10.1016/j.ymgme.2025.109178
  3. Molecules. 2025 Jun 15. pii: 2599. [Epub ahead of print]30(12):
      Phosphomannomutase 2 (PMM2) catalyzes the interconversion of mannose-6-phosphate and mannose-1-phosphate, a key step in the biosynthesis of GDP-mannose for N-glycosylation. Its deficiency is the most common cause of congenital disorders of glycosylation (CDGs), accounting for the subtype known as PMM2-CDG. PMM2-CDG is a rare autosomal recessive disease characterized by multisystemic dysfunction, including cerebellar atrophy, peripheral neuropathy, developmental delay, and coagulation abnormalities. The disease is associated with a spectrum of pathogenic missense mutations, particularly at residues involved in dimerization and catalytic function (i.e., p.Phe119Leu and p.Arg141His). The dimerization of PMM2 is considered essential for enzymatic activity, although it remains unclear whether this supports structural stability alone, or whether both subunits are catalytically active-a distinction that may affect how mutations in each monomer contribute to overall enzyme function and disease phenotype. PMM2 has a paralog, phosphomannomutase 1 (PMM1), which shares substantial structural similarity-including obligate dimerization-and displays mutase activity in vitro, but does not compensate for PMM2 deficiency in vivo. To investigate potential heterodimerization between PMM1 and PMM2 and the effect of interface mutations over PMM2 dimer stability, we first assessed the likelihood of their co-expression using data from GTEx and the Human Protein Atlas. Building on this expression evidence, we modeled all possible dimeric combinations between the two paralogs using AlphaFold3. Models of the PMM2 and PMM1 homodimers were used as internal controls and aligned closely with their respective reference biological assemblies (RMSD < 1 Å). In contrast, the PMM2/PMM1 heterodimer model, the primary result of interest, showed high overall confidence (pLDDT > 90), a low inter-chain predicted alignment error (PAE∼1 Å), and robust interface confidence scores (iPTM = 0.80). Then, we applied PISA, PRODIGY, and mmCSM-PPI to assess interface energetics and evaluate the impact of missense variants specifically at the dimerization interface. Structural modeling suggested that PMM2/PMM1 heterodimers were energetically viable, although slightly less stable than PMM2 homodimers. Interface mutations were predicted to reduce dimer stability, potentially contributing to the destabilizing effects of disease-associated variants. These findings offer a structural framework for understanding PMM2 dimerization, highlighting the role of interface stability, paralogs co-expression, and sensitivity to disease-associated mutations.
    Keywords:  PMM2-CDG; PPI; human PMMs; structural bioinformatics
    DOI:  https://doi.org/10.3390/molecules30122599
  4. Pharmaceuticals (Basel). 2025 Jun 19. pii: 921. [Epub ahead of print]18(6):
      Alzheimer's disease (AD), a progressive neurodegenerative disorder, is marked by the pathological accumulation of amyloid-β plaques and tau neurofibrillary tangles, both of which disrupt neuronal communication and function. Emerging evidence highlights the role of extracellular vesicles (EVs) as key mediators of intercellular communication, particularly in the propagation of pathological proteins in AD. Among the regulatory factors influencing EV composition and function, neuraminidase 1 (NEU1), a lysosomal sialidase responsible for desialylating glycoproteins has gained attention for its involvement in EV glycosylation. This review explores the role of NEU1 in modulating EV glycosylation, with particular emphasis on its influence on immune modulation and intracellular trafficking pathways and the subsequent impact on intercellular signaling and neurodegenerative progression. Altered NEU1 activity has been associated with abnormal glycan profiles on EVs, which may facilitate the enhanced spread of amyloid-β and tau proteins across neural networks. By regulating glycosylation, NEU1 influences EV stability, targeting and uptake by recipient cells, primarily through the desialylation of surface glycoproteins and glycolipids, which alters the EV charge, recognition and receptor-mediated interactions. Targeting NEU1 offers a promising therapeutic avenue to restore EV homeostasis and reduces pathological protein dissemination. However, challenges persist in developing selective NEU1 inhibitors and effective delivery methods to the brain. Furthermore, altered EV glycosylation patterns may serve as potential biomarkers for early AD diagnosis and monitoring. Overall, this review highlights the importance of NEU1 in AD pathogenesis and advocates for deeper investigation into its regulatory functions, with the aim of advancing therapeutic strategies and biomarker development for AD and related neurological disabilities.
    Keywords:  Alzheimer’s disease; Neuraminidase 1 (NEU1); extracellular vesicles (EVs); glycomedicine; glycosylation; intercellular communication
    DOI:  https://doi.org/10.3390/ph18060921