ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
A novel CD71 Centyrin:Gys1 siRNA conjugate reduces glycogen synthesis and glycogen levels in a mouse model of Pompe disease.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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4 citing papers in PubMed.
- Unifying the Communities of Early-Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study ofJIMD reports · 2026Article
- RNA-Based Therapies for Inherited Metabolic Disorders.Journal of inherited metabolic disease · 2026Review
- Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs.Nucleic acids research · 2025Article
- Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.Frontiers in neurologyReview
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10 authors.
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Abstract
Pompe disease is caused by acid alpha-glucosidase (GAA) deficiency, resulting in lysosomal glycogen accumulation. This disease is characterized by progressive skeletal muscle weakness, respiratory distress, and in the infantile-onset form, cardiomyopathy. The only approved treatment is enzyme replacement therapy (ERT) with human recombinant GAA. While ERT therapy extends life span, residual symptoms remain, with poor muscle uptake and immunogenicity limiting efficacy. We examined a novel Centyrin protein-short interfering ribonucleic acid (siRNA) conjugate targeting CD71 (transferrin receptor type 1, TfR1) and GYS1, a key enzyme involved in glycogen synthesis. Unlike existing ERTs designed to degrade aberrant glycogen deposits observed in Pompe patients, the CD71 Centyrin:Gys1 siRNA is designed to restore glycogen balance by inhibiting glycogen synthesis. To this end, we administered the CD71 Centyrin:Gys1 siRNA conjugate to the 6
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