Article

AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALS

2024-02-02

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is characterized by motor neuron death due to nuclear loss and cytoplasmic aggregation of the splice factor TDP-43. Pathologic TDP-43 associates with stress granules (SGs) and downregulating the SG-associated protein Ataxin-2 (Atxn2) using antisense oligonucleotides (ASO) prolongs survival in the TAR4/4 sporadic ALS mouse model, a strategy now in clinical trials. Here, we used A...

Topics

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

Identifiers and source

Literature Corpus work: 8fad6b4f-2dd2-57c6-b9f7-8e6289944536
DOI: 10.1101/2024.01.31.578314

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALSDOI 10.1101/2024.01.31.578314Read

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 miceSemantic proximity Read

Oligonucleotide therapeutics in neurodegenerative diseasesSemantic proximity Read

<i>Atxn2</i> -CAG100-KnockIn mouse spinal cord shows progressive TDP43 pathology associated with cholesterol biosynthesis suppressionSemantic proximity Read

<i>Atxn2</i> -CAG100-knock-in affects mouse lifespan and vestibulo-cerebellar function via neural disconnectionSemantic proximity Read

ATAXIN-2 intermediate-length polyglutamine expansions elicit ALS-associated metabolic and immune phenotypes.Semantic proximity Read

Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteinsSemantic proximity Read

Gain-of-function ANXA11 mutation cause late-onset ALS with aberrant protein aggregation, neuroinflammation and autophagy impairmentSemantic proximity Read

Targeted brain-specific tauopathy compromises peripheral skeletal muscle integrity and functionSemantic proximity Read

Ataxin-2 regulates RGS8 translation in a new BAC-SCA2 transgenic mouse model.Semantic proximity Read

Ataxin-2 gene: a powerful modulator of neurological disordersSemantic proximity Read

Mid-Gestation lethality of Atxn2l-Ablated MiceSemantic proximity Read

A behavioral screen for mediators of age-dependent TDP-43 neurodegeneration identifies SF2/SRSF1 among a group of potent suppressors in both neurons and gliaSemantic proximity Read