RNA Interference and Gene Delivery

RNAi Gene knockdown of R-opsin and F-actin in the marineannelid Streblospio benedicti by delivering dsRNA

This paper demonstrates RNAi gene knockdown in the marine polychaete Streblospio benedicti using dsRNA delivery via feeding larvae or microinjecting juveniles, validated by qPCR and in situ hybridization.

Cleavage of MALAT1 RNA by 14-nt sgRNA-guided tRNase ZL

This study evaluates 14-nt sgRNAs for suppressing MALAT1 RNA using tRNase ZL, showing cleavage efficiency and nuclear localization of sgRNAs.

LipiGo: A Versatile DNA-Lipid Nanoparticle Hybrid for Precision Drug Delivery

LipiGo is a DNA-lipid nanoparticle hybrid that enables efficient delivery of functional mRNA to non-hepatic tissues, particularly the spleen, through passive redistribution and active targeting.

Gastrointestinal delivery of mRNA lipid nanoparticles selectively targets the pancreas

This study shows that gastric delivery of cationic SM-102 LNPs significantly enhances pancreas targeting compared to intravenous delivery.

Intravenous lipid-siRNA conjugate mediates gene silencing at the blood-brain barrier and blood-CSF barrier

This paper demonstrates that an intravenous lipid-siRNA conjugate can deliver gene silencing to brain barriers without affecting parenchymal tissue.

Bigger Is Better Than Many: A Strategy to Optimize Multi-Gene Co-Expression

This study shows that using a single plasmid for co-transfection is more efficient and results in tighter gene expression correlation than using multiple plasmids.

Cell type distribution of intrathecal antisense oligonucleotide activity in deep brain regions of non-human primates

The paper assesses the cell-type-specific activity of intrathecally delivered antisense oligonucleotides in deep brain regions of non-human primates, finding broad target engagement across neuronal and non-neuronal cells.

Highly controllable co-delivery of siRNA and doxorubicin via cationic niosomes for synergistic anticancer effects

This study develops a co-delivery system using cationic niosomes to deliver siRNA and doxorubicin, showing enhanced anticancer effects in MDA-MB-231 cells.

Versatile Cell Penetrating Peptide for Multimodal CRISPR Gene Editing in Primary Stem Cells

This paper demonstrates that the RALA peptide effectively delivers CRISPR components in DNA, RNA, and RNP formats to primary mesenchymal stem cells with high viability and low cytotoxicity.

Poly(UG)-tailed RNAs are involved in the control of thousands of genes predominantly in the germline in Pristionchus pacificus

The paper shows poly(UG)-tailed RNAs induce gene knockdown in Pristionchus pacificus and are abundant in germline-specific genes, with mutations affecting fertility.

In silico self-assembly and complexation dynamics of cationic lipids with DNA nanocages to enhance lipofection

This study uses MD simulations to investigate how cationic lipids interact with DNA nanocages to enhance drug delivery efficiency.

Development of LYTACs via incorporating a nucleolin-targeting and lysosome-directed aptamer

This paper develops LYTACs by conjugating the nucleolin-targeting aptamer SAPT8 with other ligands to enable lysosomal degradation of target proteins in tumors and rheumatoid arthritis fibroblasts.

Cap-independent co-expression of dsRNA-sensing and NF-κB pathway inhibitors enables tunable self-amplifying RNA expression with reduced immunotoxicity

This paper develops a strategy to reduce immunotoxicity in self-amplifying RNA by encoding immune suppressors that bypass translation shutdown, enabling sustained transgene expression in mouse cells.

Organ-confined mRNA delivery and protein expression during ex vivo perfusion of porcine donor kidneys

The paper demonstrates lipid nanoparticle-mediated mRNA delivery enables rapid, organ-wide protein expression in human-sized porcine donor kidneys during normothermic machine perfusion.

Microinjected dsRNA triggers a robust RNAi response in Stentor coeruleus

This study demonstrates that microinjection of dsRNA induces a robust RNAi response in Stentor coeruleus, offering an improved method for genetic experiments when feeding-based RNAi is inadequate.

Preparation and characterization of Fe3O4-spermine-PCL-chitosan-PEG-FA nanoparticles for DNA delivery into AGS cells

This study develops and characterizes Fe3O4-spermine-PCL-chitosan-PEG-FA nanoparticles for DNA delivery into AGS cells, showing improved transfection efficiency and reduced cytotoxicity compared to PEI/DNA complexes.

AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma

This study evaluates AAV6-mediated MUC5AC siRNA delivery to reduce mucin overexpression and improve mucociliary clearance in asthma models.

Machine Learning Guided Structure Activity Discovery of Polymer Configurations in Lipid Nanoparticles for Kiss-and-Run Endosomal Escape

This paper optimizes lipid nanoparticle polymer configurations for endosomal escape using machine learning, achieving high gene editing efficiency in mice.

In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery

This paper develops an antibody-free lipid nanoparticle system for in vivo mRNA delivery to human HSCs, enabling efficient base editing of HBG to treat blood disorders like beta-thalassemia and sickle cell disease.

Structure - silencing duration relationships in RNAi medicines in rapidly dividing cells

This study examines how chemical modifications affect the duration of RNAi-based therapies in rapidly dividing cells, finding that specific modifications can extend silencing duration significantly.

Systematic feature and architecture evaluation reveals tokenized learned embeddings enhance siRNA efficacy prediction

This study evaluates features and architectures for siRNA efficacy prediction, finding that tokenized learned embeddings improve accuracy and generalizability over complex models.

Development of Emerin mRNA Lipid Nanoparticles to Rescue Myogenic Differentiation

This study develops emerin mRNA lipid nanoparticles (EMD-LNPs) to restore emerin protein expression in emerin-null myogenic progenitors, improving differentiation and showing low toxicity.

Machine Learning Reveals Intrinsic Determinants of siRNA Efficacy

This paper develops a machine learning model to predict siRNA efficacy using sequence features, achieving high accuracy and biological interpretability.

Multifunctional semiconducting polymer dots as a miRNA delivery system for osteogenic differentiation and stem cell tracking

This study develops SPM-dots as a multifunctional carrier for miRNA delivery and stem cell tracking, showing enhanced osteogenic differentiation and in vivo labeling.

Development of conditional-siRNA programmable riboswitch for targeting adverse cardiac remodeling

This paper develops a conditional-siRNA system that targets calcineurin in cardiac cells to reduce hypertrophy by responding to nppa mRNA, showing specific activation under stress conditions.

A Versatile Antibody Capture System that Drives Precise In Vivo Delivery of mRNA loaded Lipid Nanoparticles and Enhances Gene Expression

This paper introduces an antibody capture system for LNPs that enhances mRNA delivery and gene expression by enabling precise targeting to T cells with minimal off-target effects.

Growth-factor ligand functionalization enhances cellular and in vivo uptake of DNA nanodevices

This study shows that functionalizing DNA nanodevices with growth-factor ligands improves their cellular and in vivo uptake and effectiveness.

Oral bioavailability of a noncoding RNA drug, TY1, that acts on macrophages

This paper demonstrates an orally bioavailable formulation of a noncoding RNA drug, TY1, which targets macrophages and shows therapeutic benefits in two tissue injury models.

Lipid nanoparticle delivered intrabodies for inhibiting necroptosis and pyroptosis

This paper uses lipid nanoparticles to deliver mRNA encoding intrabodies that inhibit necroptosis and pyroptosis by targeting MLKL and ASC, respectively.

Nanocarrier-driven dual targeting VCAM-1/Collagen IV enables RNA interference-mediated silencing of Smad3 and Runx2 to mitigate aortic valve disease

This study develops dual-targeted nanocarriers to deliver shRNA against Smad3 and Runx2, reducing aortic valve calcification in a mouse model.