Evidence map›Paper›PMID 41244709›Full record

ReviewFrontiers in molecular biosciences2025

RNA-based therapies for neurodevelopmental disorders: innovative tools for molecular correction.

Denise Drongitis, Lucia Verrillo, Alberto de Bellis, Maria Giuseppina Miano

Abstract readReview
In one paragraph

Review in Frontiers in molecular biosciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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

1 citing paper in PubMed.

  1. Review
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

4 authors.

Denise DrongitisInstitute of Genetics and Biophysics "Adriano Buzzati-Traverso", CNR, Naples, Italy.
Lucia VerrilloInstitute of Genetics and Biophysics "Adriano Buzzati-Traverso", CNR, Naples, Italy.
Alberto de BellisMaria Rosaria Maglione Foundation Onlus, Naples, Italy.
Maria Giuseppina MianoInstitute of Genetics and Biophysics "Adriano Buzzati-Traverso", CNR, Naples, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Modulation of RNA and protein expression to restore or normalize neuronal function has emerged as a powerful therapeutic strategy for neurodevelopmental disorders (NDDs) tailoring individual genetic mutations causing intellectual disability (ID), or autism spectrum disorder (ASD), or developmental epileptic encephalopathy (DEE). In recent years, diverse classes of RNA-based molecules have been developed with therapeutic potential, including antisense oligonucleotides (ASOs), oligonucleotides targeting natural antisense transcripts (antagoNATs), Short Interspersed Nuclear Element UP-regulating RNAs (SINEUPs), interfering RNAs (RNAi), Exon-Specific engineering U1 small nuclear RNAs (ExSpeU1s), and small-activating RNA (saRNA) This review highlights the promising advances of these RNA-based therapeutics in addressing syndromic ID, such as Fragile X syndrome,

Indexed as

animal modelsgene expression regulationgenetic NDDshaploinsufficiencyRNA metabolismtranslation

Identifiers

PMID41244709
PMCPMC12615191

What OpenQuestion holds

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LicenceCC BY
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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.