RNA Interference and Gene Delivery

Naked antisense oligonucleotides access the cytosol during early endocytic trafficking

This study tracks antisense oligonucleotides (ASOs) through endocytic trafficking, finding that productive escape occurs early in endosomes before sequestration in late compartments.

AI-driven discovery and engineering of human endogenous nanocage proteins for mRNA delivery

The paper develops DeepDelivery, an AI platform that identifies human proteins for mRNA delivery, validating 80% of top candidates for efficient, non-inflammatory delivery.

Improved efficacy and tolerability of antisense oligonucleotide with Guanidine- bridged nucleic acid

This study evaluates GuNA-modified ASOs, showing improved KD activity, long-term efficacy, and reduced toxicity in a mouse model.

Synthetic Poly(2-Oxazoline) Chimera 'Lipo-Polymers' as Replacements for Key Lipids in Lipid Nanoparticles

This paper introduces synthetic polymer-based lipid alternatives for lipid nanoparticles, showing they can replace standard lipids while maintaining transfection activity.

A light-responsive lipid nanoparticle platform delivering Cas9 RNP for VEGFA editing suppresses choroidal neovascularisation

This study develops a light-responsive lipid nanoparticle platform for VEGFA gene editing to suppress choroidal neovascularisation, showing reduced vascular leakage and structural abnormalities in a rat model.

Lipid nanoparticle shape and morphology determine the magnitude and duration of mRNA transfection

This study shows that LNP shape and morphology affect mRNA transfection efficiency and duration, with non-spherical LNPs showing higher expression despite similar uptake to spherical ones.

Feed-based delivery of plasmid DNA using milk-derived extracellular vesicle-polyethylenimine complexes enables hepatic transgene expression in goldfish

PDGFRα-directed fibroblast-homing peptides for targeted delivery of nucleic acid therapeutics

The paper identifies fibroblast-homing peptides targeting PDGFR for targeted delivery of nucleic acid therapeutics, showing enhanced efficacy in a collagen VI-related muscular dystrophy model.

Cowpea Chlorotic Mottle Virus virus-like particles as DNA or RNA delivery vehicle to fish cells

This study evaluates CCMV VLPs as DNA/RNA delivery vehicles for fish cells and zebrafish, showing efficient in vivo DNA transfer to myocytes.

StoichRNA: A Scalable Framework to Map and Optimize Combinatorial RNA Systems at Single-Cell Resolution

StoichRNA maps and optimizes combinatorial RNA systems at single-cell resolution by analyzing RNA stoichiometry and identifying optimal combinations for desired cellular functions.

High efficiency gene editing and phenotype generation in avian embryos using lipid nanoparticles as a vector delivery platform

This paper demonstrates efficient gene editing and phenotype generation in avian embryos using lipid nanoparticles as a delivery platform, showing successful CRISPR/Cas9 editing and long-term labeling of cranial NCC derivatives.

Lipid nanoparticle-mediated RNAi targeting of KRAS mutants sensitises colorectal cancer cells to chemotherapy

The nuclear Argonaute HRDE-1 broadly interacts with euchromatin via an RGG domain to enable transgenerational epigenetic silencing

This study identifies the RGG domain of HRDE-1 as essential for chromatin binding and transgenerational epigenetic silencing in C. elegans, revealing a conserved mechanism for AGO-chromatin interaction.

PINPOINT enables non-destructive nanoscopic quality control of individual lipid nanoparticles and their mRNA cargo

PINPOINT provides non-destructive single-particle analysis of lipid nanoparticles and their mRNA cargo, revealing heterogeneity not detectable by bulk methods.