Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease

Large population-based studies of Parkinson's disease (PD) have identified susceptibility genes, including SLC17A5. Biallelic mutations in SLC17A5, encoding the lysosomal sialic acid transporter sialin, cause the rare neurodegenerative disease, free sialic acid storage disorder (FSASD). To expl...

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Main Authors: Marya S. Sabir, Mahin S. Hossain, Laura Pollard, Marjan Huizing, William A. Gahl, Frances M. Platt, May Christine V. Malicdan
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Biochemistry and Biophysics Reports
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Online Access:http://www.sciencedirect.com/science/article/pii/S2405580825000664
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author Marya S. Sabir
Mahin S. Hossain
Laura Pollard
Marjan Huizing
William A. Gahl
Frances M. Platt
May Christine V. Malicdan
author_facet Marya S. Sabir
Mahin S. Hossain
Laura Pollard
Marjan Huizing
William A. Gahl
Frances M. Platt
May Christine V. Malicdan
author_sort Marya S. Sabir
collection DOAJ
description Large population-based studies of Parkinson's disease (PD) have identified susceptibility genes, including SLC17A5. Biallelic mutations in SLC17A5, encoding the lysosomal sialic acid transporter sialin, cause the rare neurodegenerative disease, free sialic acid storage disorder (FSASD). To explore a potential biochemical link between FSASD and PD, we investigated ganglioside concentrations in a novel mouse model harboring the Slc17a5 p.Arg39Cys (p.R39C) variant. Our analysis revealed no significant alterations in ganglioside concentrations in heterozygous p.R39C mice, warranting further studies into other potential links between PD and sialin defects.
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series Biochemistry and Biophysics Reports
spelling doaj-art-d934fba7bbf34be295d7ec59cb3114322025-08-20T02:01:05ZengElsevierBiochemistry and Biophysics Reports2405-58082025-06-014210197910.1016/j.bbrep.2025.101979Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's diseaseMarya S. Sabir0Mahin S. Hossain1Laura Pollard2Marjan Huizing3William A. Gahl4Frances M. Platt5May Christine V. Malicdan6UDP Translational Laboratory, NIH Undiagnosed Diseases Program, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA; NIH Oxford-Cambridge Scholars Program, University of Oxford, Oxford, UKHuman Biochemical Genetics Section, Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USABiochemical Genetics Laboratory, Greenwood Genetic Center, Greenwood, SC, USAHuman Biochemical Genetics Section, Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USAUDP Translational Laboratory, NIH Undiagnosed Diseases Program, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA; Human Biochemical Genetics Section, Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USADepartment of Pharmacology, University of Oxford, Oxford, UK; Corresponding author. Department of Pharmacology, University of Oxford, UK.UDP Translational Laboratory, NIH Undiagnosed Diseases Program, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA; Human Biochemical Genetics Section, Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA; Corresponding author. National Human Genome Research Institute, National Institutes of Health, USA.Large population-based studies of Parkinson's disease (PD) have identified susceptibility genes, including SLC17A5. Biallelic mutations in SLC17A5, encoding the lysosomal sialic acid transporter sialin, cause the rare neurodegenerative disease, free sialic acid storage disorder (FSASD). To explore a potential biochemical link between FSASD and PD, we investigated ganglioside concentrations in a novel mouse model harboring the Slc17a5 p.Arg39Cys (p.R39C) variant. Our analysis revealed no significant alterations in ganglioside concentrations in heterozygous p.R39C mice, warranting further studies into other potential links between PD and sialin defects.http://www.sciencedirect.com/science/article/pii/S2405580825000664Parkinson's diseaseSalla diseaseSLC17A5SialinGlycosphingolipidsMouse model
spellingShingle Marya S. Sabir
Mahin S. Hossain
Laura Pollard
Marjan Huizing
William A. Gahl
Frances M. Platt
May Christine V. Malicdan
Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease
Biochemistry and Biophysics Reports
Parkinson's disease
Salla disease
SLC17A5
Sialin
Glycosphingolipids
Mouse model
title Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease
title_full Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease
title_fullStr Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease
title_full_unstemmed Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease
title_short Lack of significant ganglioside changes in Slc17a5 heterozygous mice: Relevance to FSASD and Parkinson's disease
title_sort lack of significant ganglioside changes in slc17a5 heterozygous mice relevance to fsasd and parkinson s disease
topic Parkinson's disease
Salla disease
SLC17A5
Sialin
Glycosphingolipids
Mouse model
url http://www.sciencedirect.com/science/article/pii/S2405580825000664
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