RNA Interference and Gene Delivery

Rapid Assessment of Immune Effector Cell-mediated Cytotoxicity using mRNA Lipid Nanoparticles

This paper introduces an mRNA-LNP method to assess immune effector cell-mediated cytotoxicity, showing it is rapid, consistent, and compatible with diverse tumor models.

HPV Capsid-Derived Cationic Peptides for Cargo Delivery and Antiviral Activity

This study identifies an HPV16-derived cationic peptide as a potent cell-penetrating peptide with antiviral activity against high-risk HPVs.

Membrane sialylation orchestrates cellular gateways: A spatiotemporal analysis of cellular transport using DNA nanocages via membrane charge modulation

The paper shows that increased cell surface sialylation enhances DNA nanocage uptake by remodeling the membrane and recruiting caveolae-mediated endocytosis in RPE1 cells.

Biodistribution of mRNA vaccines in rats: Enrichment in injection site and lymph tissues and rapid clearance without tissue persistence

Three mRNA-LNP products sharing one lipid matrix show rat intramuscular biodistribution (injection site, lymph nodes, spleen) and rapid clearance, with no Spike accumulation after repeat dosing.

Bioinspired Virus-Like Porous Silica Amplify Lipid-Mediated mRNA Delivery

This study develops hybrid lipid-VLPSi nanoparticles to enhance mRNA delivery, finding that amino-modified VLPSi significantly improves transfection efficiency compared to conventional LNPs.

Enhanced lung delivery of an immunostimulatory duplex RNA augments the antitumor activity by reshaping systemic cytokine pharmacodynamics

This study shows that lung-targeted delivery of immunostimulatory dsRNA via LNPs enhances systemic cytokine responses and antitumor activity in melanoma models.

DNA-based delivery of incretin receptor agonists using MYO Technology leads to durable weight loss in a diet-induced obesity model

Circular mRNA against Clean Cap linear mRNA vectors comprehensive comparison, expression, active and passive immunization

This study compares circular and linear mRNA vectors, finding linear vectors have higher expression levels but equal immunogenicity and protective activity.

Genome-wide screening reveals producer-cell modifications that improve virus-like particle production and delivery potency

The paper identifies genetic modifications in producer cells that enhance virus-like particle production and delivery potency.

Non-invasive Bdnf mRNA therapy improves cognition in ageing and Alzheimers mouse models

The paper demonstrates that intranasal delivery of BDNF mRNA improves memory in aged and Alzheimer's mouse models.

Single injection of modified self-amplifying RNA encoding a CD19 bispecific T cell engager mediates long-term malignant B cell clearance

This study develops a modified self-amplifying RNA-based BiTE system that provides long-term B cell clearance with a single injection.

Coordinated Tuning of Ionizable Lipids and Formulation Redirects mRNA Vaccines Toward Lymphoid-Specific CD4+ T Cell Immunity

This study shows that modifying ionizable lipids and formulation parameters of mRNA vaccines can enhance CD4+ T cell immunity and improve vaccine efficacy.

Lipid Nanoparticles for Spleen-Targeted RNA delivery

This study develops lipid nanoparticles (LNPs) for spleen-targeted RNA delivery without altering charge, showing improved splenic accumulation and distinct transcriptomic effects compared to conventional LNPs.

Rapid and reliable quantification of cytosolic mRNA escape (RNASCAPE)

RNASCAPE quantifies mRNA cytosolic escape using deep learning, achieving 78% accuracy with three timepoints and LNP parameters, revealing cholesterol's impact on mRNA release.

A Rapidly Excretable, ROS-Scavenging Ionizable Lipid Decouples mRNA Delivery Potency from Toxicity

This paper introduces a dual-function ionizable lipid that reduces mRNA toxicity and enhances clearance, achieving high gene editing efficiency in a macular degeneration model.

Theoretical estimate of the effective pKa of titratable lipids using continuum electrostatics

The paper develops a continuum electrostatics model to predict the effective pKa of ionizable lipids in lipid bilayers, showing how it shifts with salt concentration and membrane composition.

Integrating Fas-mediated apoptosis with IFNγ signaling to drive tumor regression in mRNA cancer therapeutics

This paper develops an mRNA-based therapy combining IFNγ and FasICD to induce tumor cell death and immune activation, showing significant tumor regression in mice.

Langerhans Cell-targeted mRNA Delivery: A Strategy for Dose-Sparing and Enhanced Anti-Tumor Immunity

This study develops a targeted mRNA delivery system to Langerhans cells, enhancing anti-tumor immunity while reducing required doses.

Structure-Guided Design and Dynamic Evaluation of VP4-Targeting siRNAs Against Rotavirus A

The paper designs and evaluates VP4-targeting siRNAs using computational methods to identify an optimized siRNA with improved structural compatibility and stability.

Programmable macromolecule delivery via engineered trogocytosis

This study demonstrates a programmable macromolecule delivery system using engineered trogocytosis to transfer proteins between cells, enabling genome editing and targeted cell ablation.

Systemic delivery of cationic liposome-mediated siRNA EGFR enhances therapeutic efficacy in a human colorectal cancer model

This paper develops a cationic liposome nanocarrier for siRNA delivery, showing enhanced tumor targeting and EGFR suppression in a colorectal cancer model.

Bioinspired Oxidized mRNA Lipid Nanoparticles for Ex Vivo Engineering of Chimeric Antigen Receptor Macrophages Targeting Solid Tumors

This paper develops a bioinspired oxidized lipid nanoparticle platform for mRNA-based engineering of CAR-Ms to target solid tumors, showing improved delivery and tumor-killing efficacy.

Sustained Relief of Chronic Pain via a Nav1.7_Targeting ASO_siRNA Conjugate

This paper develops an ASO-siRNA conjugate that provides sustained pain relief by targeting Nav1.7, outperforming individual components and existing drugs in preclinical models.

DNA-Directed Assembly of Multivalent Lipid Nanoparticles for Targeted T Cell Gene Delivery

This paper develops a DNA-tethering method to rapidly assemble bispecific lipid nanoparticles for targeted T cell mRNA delivery, showing improved targeting and transfection compared to single-targeted LNPs.

Sensor-mediated fine-tuning of siRNA levels is required for spermatogenic piRNA pathway function

The paper shows that sensor-mediated fine-tuning of siRNA levels is essential for proper piRNA pathway function and spermatogenesis in C. elegans.

Targeted Epigenetic Modulation Outperforms Nuclease- and Deaminase-Based Editing for Durable Pcsk9 Silencing in a Clinically Relevant Delivery System

This study compares epigenetic gene editing, base editing, and siRNA for PCSK9 suppression, finding epigenetic editing provides the most durable and efficient PCSK9 repression in mice.

Structure-Activity Mapping of Intraperitoneal mRNA-LNPs: Decoupling Tumor and Liver Biodistribution in Pancreatic Cancer

This study identifies mRNA-LNP formulation features that enhance tumor delivery and reduce liver accumulation in pancreatic cancer.

Intranasal oxytocin mRNA-LNP can promote social behaviour and reduce pain

This study demonstrates that intranasal mRNA-LNP delivery of synthetic oxytocin promotes social behavior and reduces pain without systemic inflammation or motor impairment.

Modulating backbone flexibility in hydroxamate siderophores for improved iron chelation and peptide nucleic acid (PNA) delivery into bacteria

This paper develops synthetic siderophores to deliver PNA into bacteria, showing improved iron chelation and PNA delivery compared to natural analogs.

Bleb formation induced by acidic mixing buffers improves liquid stability of mRNA-LNPs

This paper shows that using acidic mixing buffers during mRNA-LNP formation increases liquid stability by reducing mRNA degradation and extending storage life.