ArticleCell death & disease2023
Sphingomyelin 16:0 is a therapeutic target for neuronal death in acid sphingomyelinase deficiency.
Article in Cell death & disease, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 24 citations in OpenAlex.
- Egg-derived sphingomyelin induces ferroptosis in cancer cells, depending on intracellular labile iron pool levels.Biochemistry and biophysics reports · 2025Article
- Review
- Lipidomic Profiling of Red Blood Cells in the Mitochondrial Fatty Acid β-oxidation Disorder MCADD Reveals Phospholipid and Sphingolipid Dysregulation.Journal of proteome research · 2025Article
- Reelin Increases the Sphingomyelin Content of the Plasma Membrane and Affects the Surface Expression of GPI-Anchored Proteins in Hippocampal Neurons.Journal of neurochemistry · 2025Article
- Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases.Molecular genetics and metabolism · 2025Article
- Pinealectomy-Induced Melatonin Deficiency Exerts Age-Specific Effects on Sphingolipid Turnover in Rats.International journal of molecular sciences · 2025Article
- Acid sphingomyelinase modulates anxiety-like behavior likely through toll-like receptor signaling pathway.Molecular brain · 2025Article
- Environmental Temperature Variation Affects Brain Lipid Composition in Adult Zebrafish (International journal of molecular sciences · 2024Article
- The impact of sphingomyelin on the pathophysiology and treatment response to olipudase alfa in acid sphingomyelinase deficiency.Genetics in medicine open · 2024Review
- Modulation of Dietary Choline Uptake in a Mouse Model of Acid Sphingomyelinase Deficiency.International journal of molecular sciences · 2023Article
- The Long-term Lung and Respiratory Outcomes of Acid Sphingomyelinase Deficiency: A 10- and 20-year Follow-up Study.In vivo (Athens, Greece)Observational
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Authors and funding
5 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disorder caused by mutations in the SMPD1 gene encoding for the acid sphingomyelinase (ASM). While intravenous infusion of recombinant ASM is an effective treatment for the peripheral disease, the neurological complications of ASMD remain unaddressed. It has been shown that aberrantly high level of total brain sphingomyelin (SM) is a key pathological event leading to neurodegeneration. Using mice lacking ASM (ASMko), which mimic the disease, we here demonstrate that among the SM species, SM16:0 shows the highest accumulation and toxicity in ASMko neurons. By targeting lysosomes, SM16:0 causes permeabilization and exocytosis of these organelles and induces oxidative stress and cell death. We also show that genetic silencing of Ceramide Synthase 5, which is involved in SM16:0 synthesis and overexpressed in the ASMko brain, prevents disease phenotypes in ASMko cultured neurons and mice. The levels of SM16:0 in plasma also show a strong correlation with those in brain that is higher than in liver, even at early stages of the disease. These results identify SM16:0 both as a novel therapeutic target and potential biomarker of brain pathology in ASMD.
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