Article

Characterization of early markers of disease progression in the mouse model of Mucopolysaccharidosis IIIB

2023-10-09

Abstract excerpt

<title>Abstract</title> <p>Mucopolysaccharidosis (MPS) IIIB, also known as Sanfilippo Syndrome B, is a devastating childhood disease. Unfortunately, there are currently no available treatments for MPS IIIB patients. Yet, animal models of lysosomal storage diseases have been valuable tools in identifying promising avenues of treatment. Enzyme replacement therapy, gene therapy, and bone marrow transplant have all s...

Topics

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

Identifiers and source

Literature Corpus work: 267f3e36-63ec-5709-8a04-6478321af6db
DOI: 10.21203/rs.3.rs-3381843/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Characterization of early markers of disease progression in the mouse model of Mucopolysaccharidosis IIIBDOI 10.21203/rs.3.rs-3381843/v1Read

Disease pathology signatures in a mouse model of Mucopolysaccharidosis type IIIBSemantic proximity Read

Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B.Semantic proximity Read

Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome.Semantic proximity Read

Development of motor deficits in a murine model of mucopolysaccharidosis type IIIA (MPS-IIIA).Semantic proximity Read

Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology.Semantic proximity Read

Zebrafish models of Mucopolysaccharidosis IIIA, IIIB, and IIIC show hyperactivity, neuroinflammation and changes in oligodendrocyte cell stateSemantic proximity Read

Voice Abnormalities in Patients with Mucopolysaccharidosis: Can They Serve as Biological Markers for Early Diagnosis and Disease Progression?Semantic proximity Read

Neurobehavioral phenotypes of neuronopathic mucopolysaccharidosesSemantic proximity Read

Phenotypic characterisation of the Mucopolysaccharidosis Type I (MPSI) Idua-W392X mouse model reveals increased anxiety-related traits in female mice.Semantic proximity Read

Comparative profiling of white matter development in the human and mouse brain reveals volumetric deficits and delayed myelination in Angelman syndromeSemantic proximity Read

Preclinical validation of AAV9-TECPR2 gene therapy in a novel knock-in model of TECPR2-related disorderSemantic proximity Read

Cognitive impairment and progressive neuroinflammation in mucopolysaccharidosis IIIA mice expressing the R245H Sgsh variantSemantic proximity Read