ArticleScientific reports2016
Synthetic long non-coding RNAs [SINEUPs] rescue defective gene expression in vivo.
Article in Scientific reports, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
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Who cites it
35 citing papers in PubMed, 53 citations in OpenAlex.
- Revisiting LSDMCA: male lethality escape and genotype-phenotype correlations.European journal of human genetics : EJHG · 2026Article
- SINEUP-Mediated Overexpression of Endogenous α-Amylase as a Therapeutic Approach in Lafora Disease.Genes · 2026Article
- Long non-coding SINEUP RNA enhancing BDNF translation prevents dendritic atrophy following an Aβ-peptide challenge.Cellular and molecular life sciences : CMLS · 2026Article
- Decoding the modulatory role of long non-coding RNAs in embryogenesis: a comprehensive review.Molecular biology reports · 2026Review
- Internal ribosome entry sites enhance translation in trans in antisense non-coding SINEUP and circular RNAs.Nucleic acids research · 2025Article
- Retinoic Acid Induced 1 and Smith-Magenis Syndrome: From Genetics to Biology and Possible Therapeutic Strategies.International journal of molecular sciences · 2025Review
- Dysregulation of GAS5-miRNA-Mediated Signaling Pathways in Cancer Pathobiology: A Comprehensive Exploration of Pathways Influenced by this Axis.Biochemical genetics · 2025Review
- SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Molecular Mechanism of lncRNAs in Regulation of Breast Cancer Metastasis; a Comprehensive Review.Cell biochemistry and biophysics · 2025Review
- Therapeutic development approaches to treat haploinsufficiency diseases: restoring protein levels.Drug discovery today · 2024Review
- Decryption of sequence, structure, and functional features of SINE repeat elements in SINEUP non-coding RNA-mediated post-transcriptional gene regulation.Nature communications · 2024Article
- SINEUP non-coding RNA activity depends on specific N6-methyladenosine nucleotides.Molecular therapy. Nucleic acids · 2023Article
- Towards SINEUP-based therapeutics: Design of anMolecular therapy. Nucleic acids · 2022Article
- Non-Coding RNAs and Oral Cancer: Small Molecules With Big Functions.Frontiers in oncology · 2022Review
- Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Long non-coding RNAs: Biogenesis, functions, and clinical significance in gastric cancer.Molecular therapy oncolytics · 2021Review
- SINEUPs: a novel toolbox for RNA therapeutics.Essays in biochemistry · 2021Review
- Programmable technologies to manipulate gene expression at the RNA level.Current opinion in chemical biology · 2021Review
- More than causing (epi)genomic instability: emerging physiological implications of transposable element modulation.Journal of biomedical science · 2021Review
- Integrated Genomics Identifies miR-181/TFAM Pathway as a Critical Driver of Drug Resistance in Melanoma.International journal of molecular sciences · 2021Article
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
Non-coding RNAs provide additional regulatory layers to gene expression as well as the potential to being exploited as therapeutic tools. Non-coding RNA-based therapeutic approaches have been attempted in dominant diseases, however their use for treatment of genetic diseases caused by insufficient gene dosage is currently more challenging. SINEUPs are long antisense non-coding RNAs that up-regulate translation in mammalian cells in a gene-specific manner, although, so far evidence of SINEUP efficacy has only been demonstrated in in vitro systems. We now show that synthetic SINEUPs effectively and specifically increase protein levels of a gene of interest in vivo. We demonstrated that SINEUPs rescue haploinsufficient gene dosage in a medakafish model of a human disorder leading to amelioration of the disease phenotype. Our results demonstrate that SINEUPs act through mechanisms conserved among vertebrates and that SINEUP technology can be successfully applied in vivo as a new research and therapeutic tool for gene-specific up-regulation of endogenous functional proteins.
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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.