RNA Interference and Gene Delivery

Evaluating the effects of aging on biodistribution and gene silencing activity of lipid-siRNA conjugates delivered into cerebrospinal fluid

This study evaluates how aging affects the distribution and gene silencing activity of lipid-siRNA conjugates in the cerebrospinal fluid of mice.

High-throughput discovery of arginine-depleted peptides enables effective antisense delivery for Duchenne muscular dystrophy

This paper develops a high-throughput platform to discover arginine-depleted cell-penetrating peptides for improved antisense delivery in Duchenne muscular dystrophy, showing reduced toxicity and enhanced efficacy.

Stereochemical identity of lipid nanoparticles modulates protein expression via internal lipid organization

This study shows that stereochemical identity of lipid nanoparticles affects their physicochemical properties and protein expression through internal lipid organization.

Intravenously Delivered Lipid Nanoparticles Access Acute Spinal Cord Injury via Disrupted Vasculature

This study shows that intravenously delivered mRNA-lipid nanoparticles can access the injured spinal cord via disrupted blood-spinal cord barrier, achieving local transgene expression in multiple cell types.

Quantitative Assessment of shRNA Loading and Delivery Efficiency of Engineered Extracellular Vesicles

This study develops an engineered extracellular vesicle platform that improves shRNA loading and delivery efficiency, achieving robust gene silencing in multiple cell types and the mouse brain.

Exploring lipid nanoparticle design spaces using self-regulating microfluidic machines and multiplexed in vivo biodistribution

This paper develops an automated microfluidic platform to optimize lipid nanoparticle formulations and evaluates their biodistribution in mice, finding that PEtOx-based LNPs show improved structural properties and bone marrow accumulation.

Collecting Duct-Targeted Lipid Nanoparticles Deliver Pkd2 mRNA to Restore Polycystin-2 and Attenuate ADPKD

This study develops lipid nanoparticles targeting collecting duct cells to deliver Pkd2 mRNA, restoring polycystin-2 and reducing cysts in ADPKD models.

Antisense Oligonucleotides Targeting an LDLR Regulatory RNA Increase LDLR Expression and Reduce LDL-cholesterol in Vivo

This paper demonstrates that antisense oligonucleotides targeting LDLR regulatory RNAs increase LDLR expression and reduce LDL-cholesterol in a mouse model.

Elucidating the effect of a rationally designed nanostructured form-switching ASO (NaFASO) for targeting long non-coding RNA to alleviate Japanese encephalitis virus infection

This paper designs and tests a novel circular ASO (NaFASO) to target lncRNA JINR1 during JEV infection, reducing viral replication and neuronal cell death.

High-density Lipoprotein Inspired Lipid Nanoparticles for Systemic RNA delivery to the Brain

This study develops HDL-inspired lipid nanoparticles for targeted RNA delivery to the brain, showing improved BBB transport and cell-type-specific therapeutic effects in mouse models.

Targeted delivery of mRNA to the heart via extracellular vesicles or lipid nanoparticles

This study compares the efficiency of cardiac-specific extracellular vesicles and lipid nanoparticles in delivering mRNA to the heart, finding that cardiac-specific vesicles are more effective with less liver accumulation.

An Integrating Sphere Based Method for Quantifying RNA Encapsulation Efficiency in Lipid Nanoparticles without Lysis

The paper introduces a lysis-free method to quantify RNA encapsulation efficiency in lipid nanoparticles using Scatter-Free Absorption Spectroscopy, achieving high accuracy comparable to traditional methods.

Targeting lncRNA JINR1 with programmable Circular Active Nano DNAzyme (CANDe) suppresses Japanese Encephalitis Virus infection

This paper develops a circular DNAzyme platform (CANDe) that targets the lncRNA JINR1 to suppress Japanese Encephalitis Virus infection.

Systematic In Silico Off-Target Assessment of siRNAs: Integrated Tissue-Specific Scoring and Cross-Species Preclinical Model Selection with TargetSureR

TargetSureR assesses siRNA off-target risks using a seven-dimensional scoring system and enables cross-species model selection for preclinical safety evaluation.

Combinatorial screening of nanoparticles for nose-to-brain RNA delivery to modulate neuroinflammation after traumatic brain injury

The paper screens lipid nanoparticles for intranasal RNA delivery to reduce neuroinflammation after traumatic brain injury, identifying effective candidates that lower microRNA-9-5p and improve outcomes.

Direct Counting of mRNA Copies Inside Individual Lipid Nanoparticles Using In Situ Lysis and Labeling

This paper introduces a single-particle microscopy method to directly count mRNA copies in lipid nanoparticles, showing differences in mRNA copy number, size, and empty fraction between two formulations.

In vivo reprogramming of cytotoxic effector CD8+ T cells via fractalkine-conjugated mRNA-LNP

This study shows fractalkine-conjugated mRNA-LNP can target and modify CD8+ T cells in vivo, enabling transient expression of proteins like IL-2 and CD62L.

Delivery of defective interfering RNA antivirals to the lungs using hyperbranched poly(beta-amino ester) nanoparticles

This study evaluates hPBAE nanoparticles for delivering diRNA antivirals to the lungs, showing efficient uptake, nuclease resistance, and safety in golden hamsters.

Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo

This study develops a combinatorial chemistry approach to create biodegradable ionizable lipids for efficient mRNA delivery and gene editing in vivo.

In vitro-reconstituted Drosophila Arc capsids deliver gene editors to dystrophic muscle

This paper demonstrates the use of in vitro-assembled Drosophila Arc1 capsids to deliver gene editors to dystrophic muscle, achieving exon skipping and dystrophin restoration in mdx mice.

Allele-specific antisense oligonucleotide treatment rescues atad3-associated phenotype in zebrafish

This study demonstrates that allele-specific antisense oligonucleotides can rescue ATAD3A-associated developmental defects in zebrafish by reducing mutant transcripts.

A generative-AI framework for target-Specific MicroRNAs towards RNAi-based drug design

This paper introduces a generative AI framework to design target-specific siRNAs for RNAi-based drug design, showing improved specificity and reduced off-target effects compared to existing methods.

Tuning siRNA packing order in lipid nanoparticles modulates oligonucleotide functional delivery

This study shows that tuning siRNA packing order in lipid nanoparticles improves functional delivery by enhancing silencing potency through less compact packing.

Mirror-Image L-DNA Nanocubes for Stable and Targeted Multimodal Drug Delivery

This paper develops L-DNA nanocubes for targeted drug delivery, showing improved stability and therapeutic efficacy compared to D-DNA nanocubes.

Harnessing TfR1 for Cross-Species Systemic Delivery of siRNAs to Deep Brain Regions Using Single-Domain Antibodies

This paper demonstrates the use of TfR1-targeting single-domain antibodies to deliver siRNAs systemically to deep brain regions, achieving efficient knockdown at low doses.

Click Chemistry-Based Strategy for Modular Ligand Attachment to siRNAs: Toward Extrahepatic RNAi

This study develops a click chemistry-based strategy for siRNA conjugation to enhance extrahepatic RNAi, finding that bis-fatty-acid conjugates improve activity in heart and muscle while showing modality-specific limitations.

Rapid receptor internalization potentiates CD7-targeted lipid nanoparticles for efficient mRNA delivery to T cells and in vivo CAR T-cell engineering

This paper compares T-cell receptor-targeted lipid nanoparticles for mRNA delivery, finding CD7-targeting achieves highest efficiency due to rapid internalization.

Small Molecule Degradation of the microRNA-21 Precursor Rescues Pathogenic Pathways in Cellular Models of Fibrosis

This study shows that degrading the miR-21 precursor with TGP-21-RiboTAC reduces pathogenic pathways in fibrosis models by reactivating tumor suppressor genes.

Benchmarking siRNA Prediction: The Role of Representation and Validation Strategies

The paper benchmarks siRNA prediction methods, showing that rigorous cross-validation and z-curve representation improve performance and reliability.

Targeting cancer-associated cell surface RNAs with oligonucleotide-drug conjugates enables broad antitumor activity

This study develops CIRCmap to profile cell-surface and intracellular RNAs, identifies cancer-associated csRNAs, and demonstrates oligonucleotide-drug conjugates targeting these RNAs show broad antitumor activity.