A slipped-CAG DNA-binding small molecule induces trinucleotide-repeat contractions in vivo.

doi:10.1038/s41588-019-0575-8

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

Community

Back to search

Article

A slipped-CAG DNA-binding small molecule induces trinucleotide-repeat contractions in vivo.

Nature genetics - 1 Feb 2020

Nakamori Masayuki, Panigrahi Gagan B, Lanni Stella, Gall-Duncan Terence, Hayakawa Hideki, Tanaka Hana, Luo Jennifer, Otabe Takahiro, Li Jinxing, Sakata Akihiro, Caron Marie-Christine, Joshi Niraj, Prasolava Tanya, Chiang Karen, Masson Jean-Yves, Wold Marc S, Wang Xiaoxiao, Lee Marietta Y W T, Huddleston John, Munson Katherine M, Davidson Scott, Layeghifard Mehdi, Edward Lisa-Monique, Gallon Richard, Santibanez-Koref Mauro, Murata Asako, Takahashi Masanori P, Eichler Evan E, Shlien Adam, Nakatani Kazuhiko, Mochizuki Hideki, Pearson Christopher E

Abstract excerpt

In many repeat diseases, such as Huntington's disease (HD), ongoing repeat expansions in affected tissues contribute to disease onset, progression and severity. Inducing contractions of expanded repeats by exogenous agents is not yet possible. Traditional approaches would target proteins driving repeat mutations. Here we report a compound, naphthyridine-azaquinolone (NA), that specifically binds slipped-CAG DNA...

Read the complete abstract on PubMed

Topics

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

Share to Huntingtin Protein

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Corpus Striatum

Share to DNA Mismatch Repair

Share to Naphthyridines

Share to Quinolones

Share to Ribonucleases

Share to TATA-Box Binding Protein

Share to Trinucleotide Repeat Expansion

Source

Open in PubMed Open full text

Identifiers

PMID: 32060489
DOI: 10.1038/s41588-019-0575-8
PMCID: PMC7043212

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

A slipped-CAG DNA-binding small molecule induces trinucleotide-repeat contractions in vivo.PMID 32060489Read ↗

Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidenceSemantic proximity Read ↗

CAG repeat-binding small molecule improves motor coordination impairment in a mouse model of Dentatorubral-pallidoluysian atrophy.Semantic proximity Read ↗

Cas9 Nickase-Mediated Contraction of CAG/CTG Repeats <i>in Vivo</i> is Accompanied by Improvements in Huntington’s Disease PathologySemantic proximity Read ↗

FAN1 exo- not endo-nuclease pausing on disease-associated slipped-DNA repeats: A mechanism of repeat instability.Semantic proximity Read ↗

Repeat instability as the basis for human diseases and as a potential target for therapy.Semantic proximity Read ↗

FAN1 nuclease processes and pauses on disease-associated slipped-DNA repeats: Mechanism against repeat expansionsSemantic proximity Read ↗

Molecular mechanisms underlying nucleotide repeat expansion disorders.Semantic proximity Read ↗

Huntington disease update: new insights into the role of repeat instability in disease pathogenesisSemantic proximity Read ↗

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

Long somatic DNA-repeat expansion drives neurodegeneration in Huntington’s diseaseSemantic proximity Read ↗

Long somatic DNA-repeat expansion drives neurodegeneration in Huntington diseaseSemantic proximity Read ↗

Allele-selective inhibition of trinucleotide repeat genes.Semantic proximity Read ↗

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