RNA Interference and Gene Delivery

This study introduces a polycharged biodegradable nanomaterial, N1-501, that efficiently delivers mRNA in vitro and in vivo across multiple cell lines and mouse models.

The paper identifies a new class of Dicer-dependent siRNAs in C. elegans, called 23H-RNAs, produced from diverse genomic features.

The paper develops a stereopure PMO-gapmer ASO to target tau, showing improved safety and activity by controlling phosphorus stereochemistry.

This paper develops sequence-controlled PEG bottlebrush polymers for enhanced oligonucleotide delivery, showing improved cellular uptake and in vivo antisense activity through optimized chemical modifications.

This study manipulates phase-separating peptide sequences to control coacervate microdroplet properties and intracellular delivery kinetics of macromolecules.

This study develops nanoparticles that co-deliver siRNA and mRNA to simultaneously silence and restore gene/protein expression in vitro and in vivo.

This study shows that unmodified mRNA can be effectively delivered to immune cells ex vivo via a non-endosomal route without needing nucleoside modifications.

The paper identifies a peptide that enhances mRNA lipid nanoparticle delivery to CF lung epithelial cells, showing improved mRNA expression in vitro and in vivo.

This study uses mechanical oscillation to enhance endosomal escape of mRNA delivered by lipid nanoparticles, improving transfection efficiency without harming cell viability or organelle integrity.

The paper shows NRDE-3 switches small RNA partners during embryogenesis to regulate gene expression in somatic cells.

This study shows adding high concentrations of Ca2+ and Mg2+ to oligonucleotide formulations prevents acute neurotoxicity in CNS delivery without affecting efficacy.

This study uses machine learning to identify LNP composition parameters that enhance transfection efficiency across cell types and modulate cell type-preferentiality.

The paper fabricates mRNA-loaded lipid nanoparticles using SMART-MaGIC technology and evaluates their transfection efficiency and functionality in vitro.

OligoFormer improves siRNA design by integrating thermodynamic calculations, RNA-FM module, and Oligo encoder, outperforming existing methods in efficacy and off-target prediction.

siRNADesign uses a Graph Neural Network to predict siRNA efficacy by analyzing RNA sequences and interactions, achieving state-of-the-art performance in both computational and experimental settings.

This study uses TIRF imaging to examine pH-induced LNP fusion and mRNA release, showing delayed fusion onset and rapid cargo escape, with implications for optimizing mRNA delivery.

This paper develops biodegradable silica nanoparticles for efficient DNA gene delivery, showing a 16-fold increase in protein expression using linear DNA compared to circular plasmid DNA.

The paper shows guide RNA sequence affects slicing rates and conformational dynamics of the Argonaute complex, with sequence variations influencing slicing efficiency and 3'-mismatch tolerance.

This paper demonstrates reliable mRNA delivery via lipid nanoparticles to specific retinal cells in mice and human ocular tissues without significant inflammation.

This paper shows that complete substitution of nucleotides in self-amplifying RNA (saRNA) reduces interferon response and enhances expression, improving vaccine potency.

This study identifies an RNAi-mediated regulatory cascade involving ALG-3 and ALG-4 that coordinates spermatogenesis in C. elegans, particularly under heat stress.

The paper reports the structure of hSIDT1, revealing its lipid binding domain's conformational flexibility and structural features.

The paper compares cell penetration of biotherapeutics across human cell lines using a novel assay and identifies factors influencing penetration differences.

The paper shows that RNA interference selectivity in C. elegans arises from gene-specific RNA metabolism and restricted amplification, not specialized pathways.

This study develops a method to deliver proteins into cells using ionizable lipid nanoparticles by modifying protein surfaces with an anionic cloak, demonstrating efficacy in cancer cell killing and signaling inhibition.

AttSiOff improves siRNA design by predicting inhibition and off-target effects using self-attention mechanisms, achieving better accuracy and generalization than existing methods.

This study delivers hIGF1-loaded nanoparticles to the placenta of a pregnant rhesus macaque via uterine artery catheterization, showing successful placental uptake and expression of hIGF1.

This study investigates how lipid nanoparticles interact with mucus in healthy and cystic fibrosis conditions, identifying factors affecting their diffusivity for improved transmucosal delivery.

The paper demonstrates that intranasal delivery of shRNA targeting HTR2A reduces anxiety and improves memory in mice and rats.

This study shows that nanoporous surfaces enhance cellular uptake of genetic material and other molecules by inducing macropinocytosis, with optimal pore size of 400 nm.