RNA Interference and Gene Delivery

This study shows that Dicer{Delta}HEL1/wt mice exhibit increased endogenous siRNA levels and functional RNAi in vivo, providing a new model for studying mammalian canonical RNAi.

This paper develops a new mRNA-BLNP3 nanoparticle that selectively delivers mRNA to antigen-presenting cells, showing improved immune responses and reduced liver accumulation compared to previous versions.

This paper introduces a dual-targeting di-valent siRNA scaffold that can silence two genes simultaneously in the CNS of mice, demonstrating potent and programmable multi-gene modulation.

This study develops an optimized siRNA transfection protocol for human and mouse adipocyte cultures throughout differentiation, improving gene knockdown efficiency.

This paper uses machine learning to predict the effectiveness of ionizable lipids for mRNA delivery, identifying the number of outside carbons as a key factor.

DeepSipred predicts siRNA inhibition using sequence, thermodynamic, and expert knowledge features, achieving state-of-the-art performance on public datasets.

This study systematically compares siRNA delivery methods to T cells, finding lipid-conjugate-mediated delivery is most effective for both resting and activated T cells ex vivo.

This paper introduces ionizable lipids with dual ethanolamine head groups for phospholipid-free mRNA delivery, showing improved stability, efficiency, and reduced toxicity.

This paper demonstrates the effective long-distance transport and gene silencing in plants using cationized bovine serum albumin (cBSA) bound to double-stranded RNA nanocomplexes.

The paper optimizes RNAi efficiency in C. elegans PVD neurons by expressing sid-1 under the mec-3 promoter, enhancing gene knockdown effects for dendrite development and regeneration studies.

This study uses machine learning to predict the silencing activity of chemically modified siRNAs based on sequence and modification patterns, showing improved performance over existing methods.

This study shows that ARPE19 cells show reduced transfection efficiency with increasing passage number, linked to dysregulated endolysosomal trafficking.

This study shows that recombinant Ranpirnase enhances mRNA transgene expression in mice by 3-6 fold and prolongs its duration by over 85% without cytotoxicity.

This paper develops 2'-OMe/LNA oligonucleotides to selectively inhibit specific miRNA precursors, demonstrating in vitro and in vivo target selectivity for miRNA family members.

This paper demonstrates that systemically delivered OTV, a TfR-binding molecule, effectively transports ASO across the BBB, achieving widespread and sustained knockdown of target genes in the CNS of mice and non-human primates.

This paper develops a PNA-peptide conjugate targeting miRNA-21 in mouse tissues, showing improved cardiac delivery and inhibition compared to PNA alone.

This study demonstrates that systemic delivery of hABCB4 mRNA LNPs ameliorates liver fibrosis and bile phosphatidylcholine levels in a PFIC3 mouse model.

This study modifies an mRNA-LNP vaccine formula with a cationic lipid to improve safety and efficiency, showing better immunogenicity and reduced off-target effects in mice.

This paper describes a platform for generating patient-derived organoids to evaluate personalized antisense oligonucleotide therapeutics for rare diseases, showing reversal of disease phenotypes in Duchenne muscular dystrophy models.

This study demonstrates the SEND process in vivo using genetically engineered mouse models, showing potential for using SEND-VLPs as a gene therapy tool for delivering mRNAs to tissues.

The paper identifies an N-terminal region in AGO proteins that is accessible in the RNA-free state and mediates specific degradation through interaction with ATI1.

This study demonstrates that lipid nanoparticles can deliver mRNA for gene editing enzymes in utero, effectively editing multiple organs with low toxicity.

This study evaluates a synthetic mRNA encoding a hyperactive Ang1 fusion protein to prevent pulmonary vascular leakage in lung injury models.

The paper presents cryo-EM structures of AtAgo10-guide RNA complexes with and without target RNA, revealing distinct slicing mechanisms in eukaryotic AGOs compared to bacterial TtAgo.

This study develops a polymer/DNA polyplex system for CRISPR/Cas9 delivery in C. elegans, enabling the generation of transgenic and heritable mutants.

The paper develops a machine learning framework for predicting siRNA efficacy using a small dataset by applying trichotomous partitioning and random forest models, achieving better performance than linear models.

This paper demonstrates allele-specific siRNA silencing of mutant huntingtin in CNS using SNP-based targeting in vitro and in vivo.

This study shows that siRNAs targeting a chromatin-associated RNA can induce its transcriptional silencing in human cells through cis-specific base pairing.

The paper designs small interfering RNA sequences targeting SARS-CoV-2 variants using bioinformatics and deep learning, producing two databases with 62 effective sequences and 10 delivery peptide-like candidates.

This study develops GalNAc-LNPs for liver gene editing in NHPs, achieving high editing efficiency and ANGPTL3 reduction in both HoFH and wild-type models.