Evidence map›Paper›PMID 35563194›Full record

ArticleInternational journal of molecular sciences2022

Fighting the Huntington's Disease with a G-Quadruplex-Forming Aptamer Specifically Binding to Mutant Huntingtin Protein: Biophysical Characterization, In Vitro and In Vivo Studies.

Claudia Riccardi, Federica D'Aria, Filomena Anna Digilio, Maria Rosaria Carillo, Jussara Amato, Dominga Fasano, Laura De Rosa, Simona Paladino, Mariarosa Anna Beatrice Melone, Daniela Montesarchio and 1 more

Open access · goldAbstract read
In one paragraph

Article in International journal of molecular sciences, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
1.5field-weighted citation impact, top 18% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 17 citations in OpenAlex.

  1. Review
  2. Article
  3. Chronic RNA G-quadruplex Accumulation in Aging and Alzheimer's Disease.bioRxiv : the preprint server for biology · 2025
    Article
  4. Decoding Nucleotide Repeat Expansion Diseases: Novel Insights fromInternational journal of molecular sciences · 2024
    Review
  5. Article
  6. Review
  7. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 2 institutions in 2 countries.

Claudia RiccardiDepartment of Chemical Sciences, University of Naples Federico II, 80126 Napoli, Italy.ORCID 0000-0002-7182-0757
Federica D'AriaDepartment of Pharmacy, University of Naples Federico II, 80131 Napoli, Italy.
Filomena Anna DigilioResearch Institute on Terrestrial Ecosystems (IRET), UOS Naples-CNR, 80131 Napoli, Italy.ORCID 0000-0002-9342-3760
Maria Rosaria CarilloResearch Institute on Terrestrial Ecosystems (IRET), UOS Naples-CNR, 80131 Napoli, Italy.
Jussara AmatoDepartment of Pharmacy, University of Naples Federico II, 80131 Napoli, Italy.ORCID 0000-0001-6096-3544
Dominga FasanoDepartment of Molecular Medicine and Medical Biotechnology, University of Naples Federico II, 80131 Napoli, Italy.
Laura De RosaDepartment of Molecular Medicine and Medical Biotechnology, University of Naples Federico II, 80131 Napoli, Italy.
Simona PaladinoDepartment of Molecular Medicine and Medical Biotechnology, University of Naples Federico II, 80131 Napoli, Italy.ORCID 0000-0001-5332-6774
Mariarosa Anna Beatrice MeloneCenter for Rare Diseases and Inter University Center for Research in Neurosciences, Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, University of Campania Luigi Vanvitelli, 80131 Napoli, Italy.ORCID 0000-0002-7213-9277
Daniela MontesarchioDepartment of Chemical Sciences, University of Naples Federico II, 80126 Napoli, Italy.ORCID 0000-0001-6295-6911
Concetta GiancolaDepartment of Pharmacy, University of Naples Federico II, 80131 Napoli, Italy.
University of Naples Federico II · ITUniversity of Campania "Luigi Vanvitelli" · IT

Funding

Italian Association for Cancer Research IG2019 n. 23198 to C. G.Italian Association for Cancer Research IG2020 n. 25046 to D.M.
6 · The paper itself

Abstract

A set of guanine-rich aptamers able to preferentially recognize full-length huntingtin with an expanded polyglutamine tract has been recently identified, showing high efficacy in modulating the functions of the mutated protein in a variety of cell experiments. We here report a detailed biophysical characterization of the best aptamer in the series, named MS3, proved to adopt a stable, parallel G-quadruplex structure and show high nuclease resistance in serum. Confocal microscopy experiments on HeLa and SH-SY5Y cells, as models of non-neuronal and neuronal cells, respectively, showed a rapid, dose-dependent uptake of fluorescein-labelled MS3, demonstrating its effective internalization, even in the absence of transfecting agents, with no general cytotoxicity. Then, using a well-established

Indexed as

Huntington DiseaseAnimalsDisease Models, AnimalDrosophila melanogasterHuntingtin ProteinNerve Tissue ProteinsNuclear ProteinsHuntingtin ProteinNerve Tissue ProteinsNuclear ProteinsaptamersDrosophila melanogaster modelG-quadruplexHuntington’s diseasephysico-chemical characterization

Identifiers

PMID35563194
PMCPMC9101412
OpenAlexW4280648211

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.