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Record W4414993271 · doi:10.1186/s12943-025-02463-y

Advances in RNA-based cancer therapeutics: pre-clinical and clinical implications

2025· review· en· W4414993271 on OpenAlex
Yubo Yan, Shuang Liu, Jie Wen, Yunlong He, Chenyang Duan, Noushin Nabavi, Milad Ashrafizadeh, Gautam Sethi, Lubin Liu, Rong Ma

Why this work is in the frame

A frame that forgets how it found something cannot be audited. These are the routes that admitted this work.

affAt least one author lists a Canadian institution in the pinned OpenAlex snapshot.

Bibliographic record

VenueMolecular Cancer · 2025
Typereview
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicRNA Interference and Gene Delivery
Canadian institutionsUniversity of Victoria
Fundersnot available
KeywordsRNA interferencemicroRNACancerSmall interfering RNAGene silencingClinical trialImmunotherapyRNAGenetic enhancement

Abstract

fetched live from OpenAlex

Cancer therapy has been revolutionised by the emergence of RNA-based therapeutics, providing several strategies and mechanisms to regulate gene expression via messenger RNA (mRNA), small interfering RNA (siRNA), microRNAs (miRNA), antisense oligonucleotides (ASOs), and RNA aptamers. The present review highlights the recent advances in the preclinical development and clinical applications of RNA-based therapeutics, focusing on the delivery strategies, biological targets, and pharmacological optimisation, together with key clinical data. mRNA therapeutics, especially those adapted from vaccine platforms are being developed for the cancer immunotherapy and protein replacement, while siRNAs and ASOs enable highly specific gene silencing and splice correction. miRNA therapies show potential for diverse oncogenic pathway control, despite ongoing challenges in the delivery and specificity. RNA aptamers are obtaining attention as tumor-targeting agents in the drug delivery systems. Progress in lipid nanoparticles, chemical modifications, and tissue-specific delivery has improved the stability and efficacy of these agents. Early-phase clinical trials report encouraging outcomes in both solid tumours and haematologic malignancies, particularly in overcoming resistance and modulating the tumor microenvironment (TME). Although challenges remain in scalability, immune activation, and deep-tumour penetration, RNA-based strategies are advancing towards integration into clinical oncology. Continued refinement of delivery technologies and targeted trial designs will be critical for translating these therapies into effective, personalized cancer treatments. • RNA-based therapies allow for precise intervention at the genetic and molecular levels of cancer. RNA-based therapies enable targeted intervention at the genetic and molecular levels of cancer. • Distinct RNA modalities including mRNA, siRNA, miRNA, ASOs, and aptamers offer provide complementary mechanisms for tumor modulation. • Advances in delivery technologies, particularly lipid nanoparticles (LNPs), have significantly improved RNA stability, targeting, and intracellular uptake. • Clinical trials report encouraging promising efficacy and tolerability stability of RNA therapeutics in both solid tumours and haematologic malignancies. • Novel approaches such as self-amplifying RNA (saRNA) and synthetic lethality are emerging as precision strategies to address tumour heterogeneity and drug resistance. Questions • How do different types of RNA therapeutics function in cancer treatment? • What are the major challenges in delivering the delivery of RNA molecules effectively to tumor sites? • How do chemical modifications improve the performance of RNA-based drugs? • What clinical evidence supports the use of RNA therapeutics in oncology? • In what ways can RNA therapies be integrated into personalized cancer care strategies?

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Other design · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.995
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0010.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.055
GPT teacher head0.464
Teacher spread0.409 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it