Article

Reduced heparan sulfate levels in cerebrospinal fluid reflect brain neuron correction in Sanfilippo B mice

2025-04-30

Abstract excerpt

Sanfilippo B syndrome is a rare, inherited disorder of heparan sulfate catabolism. Cerebrospinal fluid heparan sulfate (HS) has been pursued as a biomarker for CNS disease. To determine whether CSF HS reflects disease in the brain, we generated a fusion protein of the NAGLU gene encoding alpha-N-acetylglucosaminidase (the enzyme deficient in Sanfilippo B) and the transmembrane region and cytosolic tail of LAMP1, w...

Topics

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

Identifiers and source

Literature Corpus work: 3e8b62de-3913-581f-a3a0-33c7c2975c8a
DOI: 10.1101/2025.04.30.651515

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Reduced heparan sulfate levels in cerebrospinal fluid reflect brain neuron correction in Sanfilippo B miceDOI 10.1101/2025.04.30.651515Read

Recombinant NAGLU-IGF2 prevents physical and neurological disease and improves survival in Sanfilippo B syndromeSemantic proximity Read

Brain transplantation of genetically corrected Sanfilippo B Neural Stem Cells induces partial cross-correction of the diseaseSemantic proximity Read

Sanfilippo Syndrome: Molecular Basis, Disease Models and Therapeutic Approaches.Semantic proximity Read

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

Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA.Semantic proximity Read

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.Semantic proximity Read

Sanfilippo syndrome: causes, consequences, and treatmentsSemantic proximity Read

Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanismsSemantic proximity Read

Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo DiseaseSemantic proximity Read

A novel iPSC model reveals selective vulnerability of neurons in Multiple Sulfatase DeficiencySemantic proximity Read

An Engineered sgsh Mutant Zebrafish Recapitulates Molecular and Behavioural Pathobiology of Sanfilippo Syndrome A/MPS IIIA.Semantic proximity Read

Heterologous HSPC transplantation rescues neuroinflammation and ameliorates peripheral manifestations in the mouse model of lysosomal transmembrane enzyme deficiency, MPS IIICSemantic proximity Read