Article

AAV-mediated gene therapy for Sialidosis

2023-11-13

Abstract excerpt

Sialidosis is a glycoprotein storage disease caused by deficiency of the lysosomal sialidase NEU1, which leads to pathogenic accumulation of sialylated glycoproteins and oligosaccharides in tissues and body fluids. The disease belongs to the group of orphan disorders with no therapy currently available. Here, we have tested the therapeutic potential of AAV-mediated gene therapy for the treatment of sialidosis in a...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: d70d6ea0-f283-5eb0-bea1-b8138ad1035f
DOI: 10.1101/2023.11.10.566667

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

AAV-mediated gene therapy for SialidosisDOI 10.1101/2023.11.10.566667Read

Genetic Insights and Clinical Implications of NEU1 Mutations in SialidosisSemantic proximity Read

Systemic and neurologic abnormalities distinguish the lysosomal disorders sialidosis and galactosialidosis in miceSemantic proximity Read

Type 1 sialidosis presenting with ataxia, seizures and myoclonus with no visual involvementSemantic proximity Read

AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy studySemantic proximity Read

Generation of novel induced pluripotent stem cell (iPSC) line from a 16-year-old sialidosis patient with NEU-1 gene mutation.Semantic proximity Read

Molecular pathology of NEU1 gene in sialidosis.Semantic proximity Read

Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage DiseaseSemantic proximity Read

Establishment and characterization of Neu1-knockout zebrafish and its abnormal clinical phenotypes.Semantic proximity Read

Generation of human induced pluripotent stem cells (hIPSCs) from sialidosis types I and II patients with pathogenic neuraminidase 1 mutations.Semantic proximity Read

Neu4, a novel human lysosomal lumen sialidase, confers normal phenotype to sialidosis and galactosialidosis cells.Semantic proximity Read

Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Semantic proximity Read

Modeling Sialidosis with Neural Precursor Cells Derived from Patient-Derived Induced Pluripotent Stem Cells.Semantic proximity Read