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

Therapeutic reversal of Huntington’s disease by in vivo self-assembled siRNAs

23 Sept 2021

Abstract excerpt

Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG expansion in exon 1 of the huntingtin (HTT) gene. Since mutant huntingtin (mHTT) protein is the root cause of Huntington's disease, oligonucleotide-based therapeutic approaches using small interfering RNAs (siRNAs) and antisense oligonucleotides designed to specifically silence mHTT may be novel therapeutic strategies for...

Read the complete abstract on PubMed

Topics

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

Share to Nerve injury and regeneration
Share to RNA Interference and Gene Delivery
Share to Tissue Engineering and Regenerative Medicine

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 34918046
DOI: 10.1093/brain/awab354

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

Therapeutic reversal of Huntington’s disease by in vivo self-assembled siRNAsPMID 34918046Read ↗

In vivo self-assembled siRNAs within small extracellular vesicles attenuate LRRK2-induced neurodegeneration in Parkinson's disease modelsSemantic proximity Read ↗

In vivo self-assembled SOD1-siRNAs mitigate muscle atrophy and denervation in amyotrophic lateral sclerosisSemantic proximity Read ↗

Delivering a disease-modifying treatment for Huntington's disease.Semantic proximity Read ↗

Efficient genome editing in the non-human primate brain using programmable extracellular vesiclesSemantic proximity Read ↗

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

Using non-coding small RNAs to develop therapies for Huntington's disease.Semantic proximity Read ↗

Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseasesSemantic proximity Read ↗

Therapeutic strategies for Huntington's diseaseSemantic proximity Read ↗

AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.Semantic proximity Read ↗

An RNA-targeting CRISPR-Cas13d system alleviates disease-related phenotypes in Huntington's disease models.Semantic proximity Read ↗

mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs <i>in vivo</i>Semantic proximity Read ↗

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

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