Evidence map›Paper›PMID 42772282›Full record

ReviewCell reports. Medicine2026

Therapeutic oligonucleotides as genetic medicines: The current state and future perspective.

Bruno Miguel Da Cruz Godinho, Chantal M Ferguson, Annemieke Aartsma-Rus

Abstract readReview
In one paragraph

Review in Cell reports. Medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Bruno Miguel Da Cruz GodinhoBoard of Directors, Oligonucleotide Therapeutics Society, San Diego, CA, USA.
Chantal M FergusonMassachusetts General Hospital, Boston, MA, USA.
Annemieke Aartsma-RusBoard of Directors, Oligonucleotide Therapeutics Society, San Diego, CA, USA; Leiden University Medical Center, Leiden, ZH, the Netherlands. Electronic address: a.m.aartsma-rus@lumc.nl.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Antisense therapeutic oligonucleotides are entering a transformative era in clinical development, with a total of 21 oligonucleotides approved and a robust pipeline poised for the coming years. These drugs have emerged as a cornerstone of genetic medicine, providing a programmable platform to modulate gene expression and treat otherwise untreatable disorders. This review covers the different mechanisms of action of these drugs and highlights the key advances in oligonucleotide chemistry and delivery that have supported the clinical successes witnessed in the last decade. It also provides a historical account and overview of the growing number of approvals and key phase 3 studies. Finally, it offers a perspective on the future of the field, initially focused only on rare diseases and now expanding to common disorders.

Indexed as

Genetic TherapyOligonucleotidesOligonucleotides, AntisenseAnimalsHumansOligonucleotidesOligonucleotides, AntisenseantisenseASOsgenetic medicineRNA interferenceRNA therapeuticssiRNAs

Identifiers

PMID42772282
PMCPMC13710550

What OpenQuestion holds

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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.