Observational studyScientific reports2021
Long non-coding RNAs as novel therapeutic targets in juvenile myelomonocytic leukemia.
Observational study in Scientific reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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Who cites it
13 citing papers in PubMed.
- Expanding the toolbox: Emerging antisense oligonucleotide mechanisms for modulating gene expression.Molecular therapy. Nucleic acids · 2026Review
- The Epigenetic Angle in the Precision Medicine Era for Blood Disorder Advancements.Sub-cellular biochemistry · 2026Review
- PCBP1-AS1 facilitates liver metastasis in pancreatic cancer by modulating miR-125b-5p in Tumor-derived exosomes to target TNFAIP3.BMC cancer · 2025Article
- MicroRNA Signatures: Illuminating Minimal Residual Disease Monitoring in Juvenile Myelomonocytic Leukemia - A Review.Journal of hematology · 2025Review
- The role of long noncoding RNAs in the diagnosis, prognosis and therapeutic biomarkers of acute myeloid leukemia.Annals of hematology · 2024Review
- Highlighting the role of long non-coding RNA (LncRNA) in multiple myeloma (MM) pathogenesis and response to therapy.Medical oncology (Northwood, London, England) · 2024Review
- The Role of Long Noncoding RNAs in Progression of Leukemia: Based on Chromosomal Location.MicroRNA (Shariqah, United Arab Emirates) · 2024Review
- BH3 mimetics and azacitidine show synergistic effects on juvenile myelomonocytic leukemia.Leukemia · 2024Article
- Biomarkers as Predictive Factors of Anti-VEGF Response.Biomedicines · 2022Review
- Article
- Molecular Mechanisms of lncRNAs in the Dependent Regulation of Cancer and Their Potential Therapeutic Use.International journal of molecular sciences · 2022Review
- Predictive Biomarkers of Age-Related Macular Degeneration Response to Anti-VEGF Treatment.Journal of personalized medicine · 2021Article
- LncRNAs serve as novel biomarkers for diagnosis and prognosis of childhood ALL.Biomarker research · 2021Article
Corrections and comments
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Authors and funding
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Juvenile myelomonocytic leukemia (JMML) treatment primarily relies on hematopoietic stem cell transplantation and results in long-term overall survival of 50-60%, demonstrating a need to develop novel treatments. Dysregulation of the non-coding RNA transcriptome has been demonstrated before in this rare and unique disorder of early childhood. In this study, we investigated the therapeutic potential of targeting overexpressed long non-coding RNAs (lncRNAs) in JMML. Total RNA sequencing of bone marrow and peripheral blood mononuclear cell preparations from 19 untreated JMML patients and three healthy children revealed 185 differentially expressed lncRNA genes (131 up- and 54 downregulated). LNA GapmeRs were designed for 10 overexpressed and validated lncRNAs. Molecular knockdown (≥ 70% compared to mock control) after 24 h of incubation was observed with two or more independent GapmeRs in 6 of them. For three lncRNAs (lnc-THADA-4, lnc-ACOT9-1 and NRIR) knockdown resulted in a significant decrease of cell viability after 72 h of incubation in primary cultures of JMML mononuclear cells, respectively. Importantly, the extent of cellular damage correlated with the expression level of the lncRNA of interest. In conclusion, we demonstrated in primary JMML cell cultures that knockdown of overexpressed lncRNAs such as lnc-THADA-4, lnc-ACOT9-1 and NRIR may be a feasible therapeutic strategy.
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