Variable responses to readthrough drugs in COL17A1 nonsense variants highlight the need for personalized therapies in junctional epidermolysis bullosa.
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Molecular therapy. Nucleic acids
Cell Press · Cell & Molecular Biology · ISSN 2162-2531
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Small extracellular vesicles carrying miR-193b-3p promote muscle regeneration by targeting AP2M1, with a significant impact observed in vitro and in vivo.
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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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Codon-dependent translation of N1-ethylpseudouridine-modified mRNA reduces innate immunity while maintaining vaccine efficacy.
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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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Cas12a chRDNA genome editing achieves near-complete target gene disruption with high specificity and no detectable off-target effects in vivo.
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Disruption of TTN uORFs does not increase titin expression in human cardiomyocytes, revealing context-dependent regulation of TTN translation.
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A novel lipid nanoparticle formulation, INTENT-2.1, effectively activates type I interferons and CD8(+) T cells to control tumors in cancer vaccines.
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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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Liver-targeted AGT siRNA increases CoRL-derived podocytes by 85% in kidney disease, promoting regeneration without immediate functional improvements.
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PCBP2 enhances miR-93-5p-mediated repression of GDF11, a tumor suppressor, promoting proliferation and invasion in hepatocellular carcinoma cells.
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miR-34a-5p acts as a radiosensitizer and biomarker, with reduced expression linked to poor treatment response and survival in esophageal adenocarcinoma.
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Engineered macrophages, including CAR-Ms, can reprogram tumor microenvironments and convert "cold" tumors into therapy-responsive niches, with over 100 genetic modifications explored.
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Adenovirus-delivered CRISPR-CasRx achieves targeted gene knockdown with minimal collateral RNA cleavage in vivo, reducing potential side effects.
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