Evidence map›Paper›PMID 34946953›Full record

ReviewGenes2021

Small Molecule Drugs Targeting Non-Coding RNAs as Treatments for Alzheimer's Disease and Related Dementias.

Lien D Nguyen, Rachel K Chau, Anna M Krichevsky

Open access · goldAbstract readReview
In one paragraph

Review in Genes, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 1 pooled it
2.4field-weighted citation impact, top 10% 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

18 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.

  1. Pooled it
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  10. [Expression relationship and significance of NEAT1 and miR-27a-3p in serum and cerebrospinal fluid of patients with Alzheimer disease].Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health sciences · 2024
    Article
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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

3 authors at 1 institution in 1 country.

Lien D NguyenDepartment of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0001-8096-1570
Rachel K ChauDepartment of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.
Anna M KrichevskyDepartment of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.
Brigham and Women's Hospital · US

Funding

Testing miR-132 signaling and replacement as a common strategy for AD, FTD, and related pathologiesR56AG069127 · NIA · BRIGHAM AND WOMEN'S HOSPITAL · PI KRICHEVSKY, ANNA M. · 2020 to 2020
$844k
Deciphering regulatory RNA networks underlying molecular pathology in Alzheimer's diseaseR21AG060019 · NIA · BRIGHAM AND WOMEN'S HOSPITAL · PI KRICHEVSKY, ANNA M. · 2018 to 2019
$492k
NIA NIH HHS R21 AG060019NIA NIH HHS R56 AG069127
6 · The paper itself

Abstract

Despite the enormous burden of Alzheimer's disease and related dementias (ADRD) on patients, caregivers, and society, only a few treatments with limited efficacy are currently available. While drug development conventionally focuses on disease-associated proteins, RNA has recently been shown to be druggable for therapeutic purposes as well. Approximately 70% of the human genome is transcribed into non-protein-coding RNAs (ncRNAs) such as microRNAs, long ncRNAs, and circular RNAs, which can adopt diverse structures and cellular functions. Many ncRNAs are specifically enriched in the central nervous system, and their dysregulation is implicated in ADRD pathogenesis, making them attractive therapeutic targets. In this review, we first detail why targeting ncRNAs with small molecules is a promising therapeutic strategy for ADRD. We then outline the process from discovery to validation of small molecules targeting ncRNAs in preclinical studies, with special emphasis on primary high-throughput screens for identifying lead compounds. Screening strategies for specific ncRNAs will also be included as examples. Key challenges-including selecting appropriate ncRNA targets, lack of specificity of small molecules, and general low success rate of neurological drugs and how they may be overcome-will be discussed throughout the review.

Indexed as

Alzheimer DiseaseDementiaDrug Evaluation, PreclinicalHumansMicroRNAsRNA, CircularRNA, Long NoncodingRNA, UntranslatedSmall Molecule LibrariesMicroRNAsRNA, CircularRNA, Long NoncodingRNA, UntranslatedSmall Molecule LibrariesAlzheimer’s disease and related dementiascircRNAdrug discoveryhigh-throughput screenslncRNAmiRNAnon-coding RNAsmall molecules

Identifiers

PMID34946953
PMCPMC8701955
OpenAlexW4200582642

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.