Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion

3 Mar 2022

Abstract excerpt

Huntington's Disease (HD) is a progressive neurodegenerative disorder caused by CAG trinucleotide repeat expansions in exon 1 of the huntingtin (HTT) gene. The mutant HTT (mHTT) protein causes neuronal dysfunction, causing progressive motor, cognitive and behavioral abnormalities. Current treatments for HD only alleviate symptoms, but cerebral spinal fluid (CSF) or central nervous system (CNS) delivery of...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Genetic Neurodegenerative Diseases
Share to Muscle Physiology and Disorders
Share to Neurogenetic and Muscular Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 35241644
DOI: 10.1038/s41467-022-28653-6

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusionPMID 35241644Read ↗

Translating Antisense Technology into a Treatment for Huntington's Disease.Semantic proximity Read ↗

<i>PMS1</i> as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington’s diseaseSemantic proximity Read ↗

Dose-Dependent Lowering of Mutant Huntingtin Using Antisense Oligonucleotides in Huntington Disease PatientsSemantic proximity Read ↗

Therapeutic antisense oligonucleotides for movement disordersSemantic proximity Read ↗

Huntington's disease: progress toward effective disease-modifying treatments and a cure.Semantic proximity Read ↗

Lowering mutant huntingtin by small molecules relieves Huntington’s disease symptoms and progressionSemantic proximity Read ↗

The Emerging Landscape of Small-Molecule Therapeutics for the Treatment of Huntington’s DiseaseSemantic proximity Read ↗

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.Semantic proximity Read ↗

Huntingtin-lowering strategies for Huntington's disease.Semantic proximity Read ↗

Anle138b ameliorates pathological phenotypes in mouse and cellular models of Huntington's disease.Semantic proximity Read ↗

Emerging therapies in Huntington’s diseaseSemantic proximity Read ↗

Anle138b ameliorates pathological phenotypes in mouse and cellular models of Huntington’s diseaseSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.