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Influenza A virus's (IAV's) frequent genetic changes challenge vaccine strategies and engender resistance to current drugs. We sought to identify conserved and essential RNA secondary structures within IAV's genome that are predicted to have greater constraints on mutation in response to therapeutic targeting. We identified and genetically validated an RNA structure (packaging stem-loop 2 (PSL2)) that mediates in vitro packaging and in vivo disease and is conserved across all known IAV isolates. A PSL2-targeting locked nucleic acid (LNA), administered 3 d after, or 14 d before, a lethal IAV inoculum provided 100% survival in mice, led to the development of strong immunity to rechallenge with a tenfold lethal inoculum, evaded attempts to select for resistance and retained full potency against neuraminidase inhibitor-resistant virus. Use of an analogous approach to target SARS-CoV-2, prophylactic administration of LNAs specific for highly conserved RNA structures in the viral genome, protected hamsters from efficient transmission of the SARS-CoV-2 USA_WA1/2020 variant. These findings highlight the potential applicability of this approach to any virus of interest via a process we term 'programmable antivirals', with implications for antiviral prophylaxis and post-exposure therapy.
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http://dx.doi.org/10.1038/s41591-022-01908-x | DOI Listing |
EMBO Mol Med
September 2025
State Key Laboratory of Pathogen and Biosecurity, Academy of Military Medical Sciences, 100071, Beijing, China.
Traditional live attenuated vaccines (LAVs) are typically developed through serial passaging or genetic engineering to introduce specific mutations or deletions. While viral RNA secondary or tertiary structures have been well-documented for their multiple functions, including binding with specific host proteins, their potential for LAV design remains largely unexplored. Herein, using Zika virus (ZIKV) as a model, we demonstrate that targeted disruption of the primary sequence or tertiary structure of a specific viral RNA element responsible for Musashi-1 (MSI1) binding leads to a tissue-specific attenuation phenotype in multiple animal models.
View Article and Find Full Text PDFMed
September 2025
Viral Research and Experimental Medicine Centre, SingHealth Duke-NUS Academic Medical Centre, Singapore 169856, Singapore; Program in Emerging Infectious Diseases, Duke-NUS Medical School, Singapore 169857, Singapore; Department of Clinical Translational Research, Singapore General Hospital, Singapo
Background: All three dengue vaccines that have completed phase 3 clinical trials have shown greater efficacy in dengue-seropositive compared to dengue-seronegative individuals. This includes the live-attenuated tetravalent dengue vaccine TAK-003, where immunogenicity in baseline seronegative individuals remains lower after two doses, despite seroconversion after the first dose, compared to baseline seropositive individuals after one dose.
Methods: A whole-genome microarray was used to analyze the host response to TAK-003.
PLoS Genet
September 2025
Neural Development Section, Mouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Frederick, Maryland, United States of America.
The RbFox RNA binding proteins regulate alternative splicing of genes governing mammalian development and organ function. They bind to the RNA sequence (U)GCAUG with high affinity but also non-canonical secondary motifs in a concentration dependent manner. However, the hierarchical requirement of RbFox motifs, which are widespread in the genome, is still unclear.
View Article and Find Full Text PDFInt J Surg Pathol
September 2025
Department of Pathology, The Thirteenth People's Hospital of Chongqing, Chongqing, China.
Soft tissue sarcomas are a heterogeneous group of malignancies arising from mesenchymal cells. Recent advancements in genomic profiling have identified novel gene fusions in these tumors, offering new insights into their pathogenesis and potential therapeutic targets. Here, we describe a spindle cell sarcoma harboring a novel gene fusion.
View Article and Find Full Text PDFJCI Insight
September 2025
Division of Nephrology, Boston University Chobanian & Avedisian School of Medicine, Boston, United States of America.
Background: Active vitamin D metabolites, including 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D (1,25D), have potent immunomodulatory effects that attenuate acute kidney injury (AKI) in animal models.
Methods: We conducted a phase 2, randomized, double-blind, multiple-dose, 3-arm clinical trial comparing oral calcifediol (25D), calcitriol (1,25D), and placebo among 150 critically ill adult patients at high-risk of moderate-to-severe AKI. The primary endpoint was a hierarchical composite of death, kidney replacement therapy (KRT), and kidney injury (baseline-adjusted mean change in serum creatinine), each assessed within 7 days following enrollment using a rank-based procedure.