Evidence map›Paper›PMID 40045578›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2025

Clinical applications of oligonucleotides for cancer therapy.

Vittorio DeFranciscis, Giovanni Amabile, Marcin Kortylewski

Abstract readReview
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing 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

18 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Article
  5. Review
  6. Article
  7. Review
  8. CRISPR Interference to Inhibit Oncogenes for Cancer Therapy.International journal of molecular sciences · 2026
    Review
  9. Review
  10. Review
  11. Article
  12. Article
  13. Review
  14. Drug Discovery Strategies for Kallikrein-Related Peptidases.International journal of molecular sciences · 2025
    Review
  15. Review
  16. From Hypoxia to Bone: Reprogramming the Prostate Cancer Metastatic Cascade.International journal of molecular sciences · 2025
    Review
  17. Review
  18. 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

3 authors.

Vittorio DeFranciscisNational Research Council, Institute of Genetic and Biomedical Research, Milan, Italy.
Giovanni AmabileAptaDiR Therapeutics, 20121 Milan, Italy.
Marcin KortylewskiDepartment of Immuno-Oncology, Beckman Research Institute at City of Hope National Medical Center, Duarte, CA 91010, USA. Electronic address: mkortylewski@coh.org.

Funding

Myeloid cell-selective, oligonucleotide-based STAT3 inhibition combined with total marrow and lymphoid irradiation for immunotherapy of acute myeloid leukemiaR01CA284593 · NCI · BECKMAN RESEARCH INSTITUTE/CITY OF HOPE · PI Susanta K Hui, Marcin Kortylewski · 2023 to 2026
$2.8M
NCI NIH HHS R01 CA284593
6 · The paper itself

Abstract

Oligonucleotide therapeutics (ONTs) represent a rapidly evolving modality for cancer treatment, capitalizing on their ability to modulate gene expression with high specificity. With more than 20 nucleic acid-based therapies that gained regulatory approval, advances in chemical modifications, sequence optimization, and novel delivery systems have propelled ONTs from research tools to clinical realities. ONTs, including siRNAs, antisense oligonucleotides, saRNA, miRNA, aptamers, and decoys, offer promising solutions for targeting previously "undruggable" molecules, such as transcription factors, and enhancing cancer immunotherapy by overcoming tumor immune evasion. The promise of ONT application in cancer treatment is exemplified by the recent FDA approval of the first oligonucleotide-based treatment to myeloproliferative disease. At the same time, there are challenges in delivering ONTs to specific tissues, mitigating off-target effects, and improving cellular uptake and endosomal release. This review provides a comprehensive overview of ONTs in clinical trials, emerging delivery strategies, and innovative therapeutic approaches, emphasizing the role of ONTs in immunotherapy and addressing hurdles that hinder their clinical translation. By examining advances and remaining challenges, we highlight opportunities for ONTs to revolutionize oncology and enhance patient outcomes.

Indexed as

Genetic TherapyNeoplasmsOligonucleotidesAnimalsClinical Trials as TopicDrug Delivery SystemsHumansImmunotherapyOligonucleotides, AntisenseOligonucleotidesOligonucleotides, Antisenseantisense oligonucleotidesaptamerscancer therapydeliveryimmunotherapyoligonucleotidesiRNA

Identifiers

PMID40045578
PMCPMC12172192

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.