Article

Antisense therapy in a new rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment

2021-01-31

Abstract excerpt

Alexander disease (AxD) is a devastating leukodystrophy caused by gain of function mutations in GFAP , and the only available treatments are supportive. Recent advances in antisense oligonucleotide (ASO) therapy have demonstrated that transcript targeting can be a successful strategy for human neurodegenerative diseases amenable to this approach. We have previously used mouse models of AxD to show that Gfap -tar...

Topics

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

Identifiers and source

Literature Corpus work: 838b1588-9d3c-5bb7-9a6c-e1af6999a009
DOI: 10.1101/2021.01.29.428244

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Antisense therapy in a new rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairmentDOI 10.1101/2021.01.29.428244Read

Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.Semantic proximity Read

Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease.Semantic proximity Read

Suppression of GFAP toxicity by alphaB-crystallin in mouse models of Alexander disease.Semantic proximity Read

Gfap Mutation and Astrocyte Dysfunction Lead to a Neurodegenerative Profile with Impaired Synaptic Plasticity and Cognitive Deficits in a Rat Model of Alexander Disease.Semantic proximity Read

Alexander disease: models, mechanisms, and medicine.Semantic proximity Read

Synergistic Effects of Glial Fibrillary Acidic Protein Mutation and Overexpression in the Pathogenesis of Alexander Disease.Semantic proximity Read

Alexander Disease: A Literature Review for Clinicians.Semantic proximity Read

Pexidartinib treatment in Alexander disease model mice reduces macrophage numbers and increases glial fibrillary acidic protein levels, yet has minimal impact on other disease phenotypes.Semantic proximity Read

Deficits in adult neurogenesis, contextual fear conditioning, and spatial learning in a Gfap mutant mouse model of Alexander disease.Semantic proximity Read

Alexander disease: a review and the gene.Semantic proximity Read

[Clinical characteristics and diagnostic criteria on Alexander disease].Semantic proximity Read

Mutations in GFAP, encoding glial fibrillary acidic protein, are associated with Alexander disease.Semantic proximity Read