RNA Interference and Gene Delivery

siRNA-based Therapeutic Candidate Targeting PRDM2 for Inhibition of Lung Cancer Progression

This study identifies ARIZ-047, an siRNA targeting PRDM2/RIZ2, as a therapeutic candidate that inhibits lung cancer progression by increasing RIZ1 expression and suppressing Wnt signaling.

Lymphatic endothelial cell-targeting lipid nanoparticles delivering VEGFC mRNA improve lymphatic function after injury

This study develops a lymphatic endothelial cell-targeting lipid nanoparticle for mRNA delivery, improving lymphatic function after injury.

Unveiling the potential of neuron-targeted dendriplexes for siRNA delivery using a PNS-CNS-on-Chip

This study develops neuron-targeted dendriplexes for siRNA delivery, showing enhanced axonal growth and CNS targeting in a PNS-CNS-on-Chip model.

An inherent T cell-activating mRNA delivery platform for in vivo CAR T generation

This paper develops a T cell-activating mRNA delivery platform that enables in vivo CAR T cell generation without exogenous stimulation or antibody conjugation.

Argonaute-siRNA loading via the RNA-binding protein RDE-4 in C. elegans

This study identifies RDE-4 as a key factor in loading siRNAs into RDE-1 and 26G-RNAs into ERGO-1 in C. elegans, revealing its role in small RNA loading specificity.

Engineering temporal alignment of mRNA delivery enables functional rescue across genetic infertility models

This study identifies temporal mismatch between mRNA delivery and spermatogenesis as a barrier to functional rescue, showing that sustained expression aligned with developmental timing enables recovery in genetic infertility models.

Lipid monolayers adsorbing mRNA as models for mRNA enclosed in lipid nanoparticles for transfection

This study uses lipid monolayers to model mRNA-lipid nanoparticle interactions, revealing how ions affect mRNA binding and electrostatic balance at the interface.

Anti-CD38-Targeted Piperazine-Derived Lipid Nanoparticles Overcome Hepatic Clearance for mRNA Delivery to Multiple Myeloma Cells In Vivo

This study develops anti-CD38 lipid nanoparticles for targeted mRNA delivery to multiple myeloma cells, improving tumor targeting and reducing hepatic accumulation.

Hydroxychloroquine decouples reactogenicity from efficacy in mRNA and viral-vector gene delivery

This study shows hydroxychloroquine reduces inflammation from mRNA and viral-vector therapies without affecting their therapeutic effectiveness.

Modular platform for therapeutic drug delivery using trifunctional bio-orthogonal macromolecular conjugates

The paper develops a modular platform for synthesizing bio-orthogonal macromolecular conjugates (BMCs) to enable targeted delivery of macromolecular therapeutics with improved efficiency and specificity.

RG motifs promote piRNA-mediated gene silencing in C. elegans

This study identifies RG motifs in C. elegans PRG-1 as critical for linking piRNA-mediated silencing to siRNA production, impacting fertility and germline maintenance.

Effects of mixing technique and ethanol removal on lipidnanoparticle physicochemical properties

This study examines how mixing techniques and ethanol removal affect lipid nanoparticle (LNP) physicochemical properties using SAXS and computational fluid dynamics.

Identifying differential effects from eleven mixing techniques on mRNA lipid nanoparticle physicochemistry and biological performance

The paper compares the effects of eleven mixing techniques on mRNA lipid nanoparticle physicochemistry and biological performance, finding significant differences in particle properties and performance across methods.

Targeting dendritic cells with RNA-loaded nanoparticles grafted with short peptides

This paper designs and evaluates RNA-loaded nanoparticles conjugated with short peptides to selectively target dendritic cells via Clec9A, improving immunotherapeutic selectivity and efficacy.

Delivery of collagen-encoding mRNA to the skin for cosmetic applications

The paper develops a method to deliver collagen-encoding mRNA topically to improve skin condition by enabling in vivo collagen expression.

Modified self-amplifying RNA mediates robust and prolonged gene expression in the mouse and ex vivo human brain

This paper demonstrates that modified self-amplifying RNA (saRNA) enables long-lasting protein expression in mouse and human brain tissues.

Elucidating lipid nanoparticle properties and structure through biophysical analyses

This study uses biophysical methods to characterize lipid nanoparticle properties and predict their transfection efficiency.

In vivo mRNA delivery to the lung vascular endothelium by dicationic Charge-Altering Releasable Transporters

This study develops dicationic Charge-Altering Releasable Transporters (CARTs) that deliver mRNA specifically to lung endothelial cells via intravenous administration.

Preclinical evaluation of Targeted IL-1β Knockdown via CD44-Immunoliposomes: A Nano-therapy against the Inflammatory Microenvironment

This paper evaluates siIL-1β-anti-CD44-Liposomes as an anti-inflammatory therapy, showing reduced pro-inflammatory cytokines and increased anti-inflammatory cytokines in macrophages.

Anionic lipids modulate mRNA-lipid nanoparticle immunogenicity and confer protection in a mouse model of multiple sclerosis

This study shows that anionic lipids in mRNA-LNPs can modulate immune responses, reducing inflammation and disease in a multiple sclerosis mouse model.

Reprogrammable bacterial nanosyringes for RNA and gene editors delivery

This paper develops DART, a bacterial nanosyringe system for delivering RNA and gene editors, demonstrating its ability to deliver various RNAs and knockout GFP in vitro and in vivo.

shRNAI: a deep neural network for the design of highly potent shRNAs

shRNAI is a deep learning model that designs potent shRNAmir gRNAs by incorporating sequence and contextual features, outperforming existing methods on both simulated and experimental data.

Targeted inhibition of colorectal carcinoma using a designed CEA-binding protein to deliver TCF/LEF transcription factor decoy DNA and p53 protein

This paper develops a targeted therapy for colorectal carcinoma using a CEA-binding protein to deliver transcription factor decoy DNA and p53 protein, showing suppression of tumor growth.

Disulfide cross-linked redox-sensitive peptide condensates are efficient cell delivery vehicles of molecular cargo.

This study demonstrates that disulfide-cross-linked peptide condensates efficiently deliver various molecular cargos, including DNA and mRNA, into HeLa cells with higher transfection efficiency than commercial agents.

IGF1R-Targeted Delivery of a Bridged Nucleic Acid Oligonucleotide-Peptide Conjugate for MicroRNA 21 Inhibition in Triple-Negative Breast Cancer

This study develops an IGF1R-targeted miR-21 inhibitor conjugate that reduces TNBC cell proliferation, induces apoptosis, and shows tumor-specific efficacy in mice with minimal toxicity.

Protein corona formed on lipid nanoparticles compromises delivery efficiency of mRNA cargo

A 16-hour continuous iodixanol density gradient workflow isolates lipid nanoparticle protein coronas from human plasma and shows that vitronectin and C-reactive protein coronas impair mRNA delivery in liver cells.

Multi-Scale Kinetics Modeling and Advanced Assay for mRNA-Lipid Nanoparticle Potency Assessment

The paper develops a multi-scale kinetic model and advanced assays to assess mRNA-LNP potency by integrating mechanistic understanding with multi-omics data.

Multifunctional PolySpermine-based Nanocapsules for Targeted Gene Delivery to Gastric Cancer Cells

This paper develops and evaluates multifunctional nanocapsules for targeted gene delivery to gastric cancer cells, showing improved biocompatibility and transfection efficiency compared to traditional vectors.

Hydroxychloroquine-functionalized Ionizable Lipids Mitigate Inflammatory Responses in mRNA Therapeutics

This study introduces HCQ-functionalized LNPs that reduce inflammation while maintaining mRNA delivery efficiency, showing promise for repeated dosing applications.

Cryopreservation of siRNA-treated cells is feasible

This study shows that cryopreservation of siRNA-treated cells maintains silencing efficacy, with optimal siRNA exposure duration and temperature.