ReviewCurrent pharmaceutical biotechnology2018
Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma.
Review in Current pharmaceutical biotechnology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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.
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.
Who cites it
31 citing papers in PubMed, 47 citations in OpenAlex.
- Designing nucleic acid-based therapeutics for cancer treatment: Updates on the state of the art.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Review
- RNA activation as a precision dosing modality: MTL-CEBPA for controlled enzyme elevation in MPS I-H.Frontiers in medicine · 2026Article
- Nanotechnology-Enabled Diagnosis and Treatment of Hepatocellular Carcinoma: Theranostics, Combination Regimens, and Translation.International journal of nanomedicine · 2026Review
- Advances and challenges of targeting epicardial adipose tissue (EAT) and perivascular adipose tissue (PVAT).Cardiovascular diabetology · 2025Review
- RNAa: Mechanisms, therapeutic potential, and clinical progress.Molecular therapy. Nucleic acids · 2025Review
- Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.Signal transduction and targeted therapy · 2025Review
- Nanocarriers: Exploring the Potential of Oligonucleotide Delivery.Current drug delivery · 2025Review
- Why Is Wnt/β-Catenin Not Yet Targeted in Routine Cancer Care?Pharmaceuticals (Basel, Switzerland) · 2024Review
- Advancements in clinical RNA therapeutics: Present developments and prospective outlooks.Cell reports. Medicine · 2024Review
- RNA-based Therapeutics: Past, Present and Future Prospects, Challenges in Cancer Treatment.Current pharmaceutical biotechnology · 2024Review
- Non-Coding RNAs: Foes or Friends for Targeting Tumor Microenvironment.Non-coding RNA · 2023Review
- Article
- Modulating the expression of tumor suppressor genes using activating oligonucleotide technologies as a therapeutic approach in cancer.Molecular therapy. Nucleic acids · 2023Review
- Lipid-based nucleic acid therapeutics with in vivo efficacy.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2023Review
- The improving strategies and applications of nanotechnology-based drugs in hepatocellular carcinoma treatment.Frontiers in bioengineering and biotechnology · 2023Review
- Emerging nanobiotechnology for precise theranostics of hepatocellular carcinoma.Journal of nanobiotechnology · 2022Review
- RNA-based therapeutics: an overview and prospectus.Cell death & disease · 2022Review
- Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria.British journal of clinical pharmacology · 2022Review
- RNA-based therapeutics to treat human fungal infections.Trends in microbiology · 2022Review
- Small activating RNA activation of ATOH1 promotes regeneration of human inner ear hair cells.Bioengineered · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundOligonucleotide drug development has revolutionised the drug discovery field. Within this field, 'small' or 'short' activating RNAs (saRNA) are a more recently discovered category of short double-stranded RNA with clinical potential. saRNAs promote transcription from target loci, a phenomenon widely observed in mammals known as RNA activation (RNAa).
objectiveThe ability to target a particular gene is dependent on the sequence of the saRNA. Hence, the potential clinical application of saRNAs is to increase target gene expression in a sequence-specific manner. saRNA-based therapeutics present opportunities for expanding the "druggable genome" with particular areas of interest including transcription factor activation and cases of haploinsufficiency. RESULTS AND
conclusionIn this mini-review, we describe the pre-clinical development of the first saRNA drug to enter the clinic. This saRNA, referred to as MTL-CEBPA, induces increased expression of the transcription factor CCAAT/enhancer-binding protein alpha (CEBPα), a tumour suppressor and critical regulator of hepatocyte function. MTL-CEBPA is presently in Phase I clinical trials for hepatocellular carcinoma (HCC). The clinical development of MTL-CEBPA will demonstrate "proof of concept" that saRNAs can provide the basis for drugs which enhance target gene expression and consequently improve treatment outcome in patients.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.