Molecular therapy. Nucleic acids
Cell Press · Cell & Molecular Biology · ISSN 2162-2531
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Latest in Mol Ther Nucleic Acids
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Erratum clarifies that overexpression of lncRNA EPB41L4A-AS1 induces metabolic reprogramming in trophoblast cells and placenta tissue of miscarriage cases.
- Open access
Fifth-generation GD2-CAR T cells secreting anti-PD-L1 scFv show superior cytotoxicity against GD2-low retinoblastoma cells at low effector-to-target ratios.
- Open access
DNA oligonucleotides encoding suppressor tRNA induce PTC readthrough in cultured cells without additional promoters.
- Open access
AptViralDB provides a comprehensive, manually curated database of 1,768 experimentally validated antiviral aptamers targeting 40 viral species and 104 molecular targets.
- Open access
Apt-E-36-3, a 30-mer DNA aptainhibitor, binds the SARS-CoV-2 E protein with nanomolar affinity and blocks its oligomerization, offering a potential therapeutic approach.
- Open access
Aptamer-guided delivery of miR-126 significantly reduces glioblastoma stem-like cell invasion and tumor progression in mouse models.
- Open access
Meiotic cohesin complexes, including REC8 and STAG3, are expressed in embryonic stem cells and influence chromatin architecture and gene regulation.
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rAAV-mediated delivery of multiple miRNAs targeting MAPK signaling suppresses laser-induced choroidal neovascularization in mice by reducing CNV leakage and angiogenesis.
- Open access
A BAC-based non-infectious SARS-CoV-2 replicon identified the M gene as a promising antisense target with an EC50 of 6.3 nM for antiviral development.
- Open access
DLL3 is essential for glioma cell viability and proliferation, with its depletion suppressing cell cycle pathways and inducing broad transcriptional reprogramming in glioma.
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Segmented poly(A) tails with microRNA target sites enable tissue-specific regulation of mRNA expression, with suppression observed in non-target cells in vitro and in vivo.
- Open access
Small extracellular vesicle-delivered miR-193b-3p enhances muscle regeneration by suppressing AP2M1 in vitro and in a murine muscle loss model.
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Multi-cycle reagent recycling in in vitro transcription and purification reduces raw-material costs by over twofold while maintaining RNA quality over five cycles.
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Variable responses to readthrough drugs in COL17A1 nonsense variants suggest personalized therapy for junctional epidermolysis bullosa.
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Codon-dependent translation of N1-ethylpseudouridine-modified mRNA reduces innate immunity while maintaining vaccine efficacy.
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Cas12a chRDNA genome editing achieves near-complete target gene disruption with high specificity and no detectable off-target effects in vivo.
- Open access
A lipid nanoparticle mRNA vaccine targeting Plasmodium vivax E140 antigen achieves up to 78% reduction in parasite invasion in human reticulocytes.
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Lipid nanoparticle delivery of mRNA achieves 100% higher T cell viability and enhanced protein expression compared to electroporation in serial human T cell engineering.
- Open access
Disruption of TTN uORFs does not increase titin expression in human cardiomyocytes, revealing context-dependent regulation of TTN translation.
- Open access
Gene therapy has achieved clinical breakthroughs for hereditary hematological disorders, with approved treatments for hemoglobinopathies and hemophilia, moving toward curative potential.
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INTENT-2.1 lipid nanoparticle induces tissue destruction and enhances anti-tumor CD8+ T cell responses via type I interferons in a cancer vaccine model.
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Topics moving, week to 23 Oct 2026
- Single-cell and spatial transcriptomics6
- Neuroinflammation and Neurodegeneration Mechanisms5
- Bacteriophages and microbial interactions5
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- Alzheimer's disease research and treatments3
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