ArticleEMBO molecular medicine2021
Alleviation of a polyglucosan storage disorder by enhancement of autophagic glycogen catabolism.
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Article in EMBO molecular medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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22citing papers in PubMed
4.9field-weighted citation impact, top 4% of its field
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Who cites it
22 citing papers in PubMed, 35 citations in OpenAlex.
- Autolysosomal enhancer GHF201 attenuates pathophysiology in a mucopolysaccharidosis type I mouse model.Life science alliance · 2026Article
- Dysregulation of glycogen metabolism in cardiomyopathy caused by glycogenin-1 (GYG1) missense variants.The Journal of clinical endocrinology and metabolism · 2026Article
- Unifying the Communities of Early-Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study ofJIMD reports · 2026Article
- Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies.Annals of neurology · 2025Review
- Splice-modulating antisense oligonucleotides targeting a pathogenic intronic variant in adult polyglucosan body disease correct mis-splicing and restore enzyme activity in patient cells.Nucleic acids research · 2025Article
- In depth profiling of dihydrolipoamide dehydrogenase deficiency in primary patients fibroblasts reveals metabolic reprogramming secondary to mitochondrial dysfunction.Molecular genetics and metabolism reports · 2025Article
- Clinical genetic analysis of an adult polyglucosan body disease (APBD) family caused by the compound heterozygous variant ofFrontiers in genetics · 2025Article
- Progressive liver disease and dysregulated glycogen metabolism in murine GSD IX γ2 models human disease.Molecular genetics and metabolism · 2024Article
- Neurological glycogen storage diseases and emerging therapeutics.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- Mitochondrial Dysfunction in Glycogen Storage Disorders (GSDs).Biomolecules · 2024Review
- The Autophagic Activator GHF-201 Can Alleviate Pathology in a Mouse Model and in Patient Fibroblasts of Type III Glycogenosis.Biomolecules · 2024Article
- Natural history study of hepatic glycogen storage disease type IV and comparison to Gbe1ys/ys model.JCI insight · 2024Article
- Amylopectinosis of the fatal epilepsy Lafora disease resists autophagic glycogen catabolism.EMBO molecular medicine · 2024Article
- Glycogen storage diseases: An update.World journal of gastroenterology · 2023Review
- Vutiglabridin Modulates Paraoxonase 1 and Ameliorates Diet-Induced Obesity in Hyperlipidemic Mice.Biomolecules · 2023Article
- Proteomic investigations of adult polyglucosan body disease: insights into the pathobiology of a neurodegenerative disorder.Frontiers in neurology · 2023Article
- Crosstalk between Glycogen-Selective Autophagy, Autophagy and Apoptosis as a Road towards Modifier Gene Discovery and New Therapeutic Strategies for Glycogen Storage Diseases.Life (Basel, Switzerland) · 2022Review
- Pathomorphogenesis of Glycogen-Ground Glass Hepatocytic Inclusions (Polyglucosan Bodies) in Children after Liver Transplantation.International journal of molecular sciences · 2022Article
- Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders.Bioengineering (Basel, Switzerland) · 2022Review
- The role of autophagy in death of cardiomyocytes.Journal of molecular and cellular cardiology · 2022Review
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Authors and funding
22 authors at 5 institutions in 2 countries.
Or KakhlonDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.ORCID 0000-0001-7435-6454
Hilla VakninLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.ORCID 0000-0001-9101-1423
Kumudesh MishraDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.ORCID 0000-0002-7537-8070
Jeevitha D'SouzaLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.
Monzer MarisatLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.
Uri SprecherLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.
Shane Wald-AltmanLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.
Anna DukhovnyLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.
Yuval RavivLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.
Benny Da'adooshBlavatnik Center for Drug Discovery, Tel Aviv University, Tel Aviv, Israel.
Hamutal EngelBlavatnik Center for Drug Discovery, Tel Aviv University, Tel Aviv, Israel.
Sandrine BenhamronDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Keren NitzanDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Sahar SweetatDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Anna PermyakovaObesity and Metabolism Laboratory, Institute for Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Anat MordechaiDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Hasan Orhan AkmanDepartment of Neurology, Columbia University Medical Center, New York, New York, USA.
Hanna RosenmannDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Alexander LossosDepartment of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Joseph TamObesity and Metabolism Laboratory, Institute for Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.ORCID 0000-0002-0948-0093
Berge A MinassianDivision of Neurology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Miguel WeilLaboratory for Neurodegenerative Diseases and Personalized Medicine, The Cell Screening Facility for Personalized Medicine, The Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Sagol School of Neurosciences, Tel Aviv University, Tel Aviv, Israel.ORCID 0000-0001-5721-653X
Tel Aviv University · ILHadassah Medical Center · ILHebrew University of Jerusalem · ILColumbia University Irving Medical Center · USThe University of Texas Southwestern Medical Center · US
Funding
Suppressing glycogen storage with small molecule inhibitors as a therapeutic approach to Lafora DiseaseP01NS097197 · NINDS · UNIVERSITY OF KENTUCKY · PI GAO, TIANYAN · 2016 to 2021
$9.0MAbstract
This work employs adult polyglucosan body disease (APBD) models to explore the efficacy and mechanism of action of the polyglucosan-reducing compound 144DG11. APBD is a glycogen storage disorder (GSD) caused by glycogen branching enzyme (GBE) deficiency causing accumulation of poorly branched glycogen inclusions called polyglucosans. 144DG11 improved survival and motor parameters in a GBE knockin (Gbe
Indexed as
Glycogen Storage DiseaseNervous System DiseasesAnimalsGlucansGlycogenMiceGlucansGlycogenpolyglucosanadult polyglucosan body diseaseautophagyglycogenlysosomespolyglucosan
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LicenceCC BY
Read underepoch 390
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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.