RNA Interference and Gene Delivery

Engineering ionizable lipids for rapid biodegradation balances mRNA vaccine efficacy and tolerability

This study designs and tests biodegradable ionizable lipids for mRNA vaccines, showing they balance efficacy and tolerability by reducing inflammation while maintaining strong immune responses.

Preliminary attempt to inhibit proliferation of NUT carcinoma cell lines using antisense oligonucleotides targeting NUTM1

This pilot study tested antisense oligonucleotides targeting NUTM1 in NUT carcinoma cell lines but found no selective inhibition, highlighting the need for further ASO optimization.

Peri-centrosomal localization of small interfering RNAs in C. elegans

The paper identifies peri-centrosomal localization of siRNAs in C. elegans using NRDE-3 as a reporter, showing dependence on 22G siRNAs and centrosomal components.

Lipid nanoparticle-mediated delivery of mRNA into the mouse and human retina and other ocular tissues.

This paper demonstrates reliable mRNA delivery via lipid nanoparticles to specific retinal cells in mice and human ocular tissues without significant inflammation.

RNAi-mediated regulation of alg-3 and alg-4 coordinates the spermatogenesis developmental program in C. elegans

This study identifies an RNAi-mediated regulatory cascade involving ALG-3 and ALG-4 that coordinates spermatogenesis in C. elegans, particularly under heat stress.

Comparing cell penetration of biotherapeutics across human cell lines

The paper compares cell penetration of biotherapeutics across human cell lines using a novel assay and identifies factors influencing penetration differences.

Target-specific requirements for RNA interference can arise through restricted RNA amplification despite the lack of specialized pathways

The paper shows that RNA interference selectivity in C. elegans arises from gene-specific RNA metabolism and restricted amplification, not specialized pathways.

A Dose-Response Model for Accurate Detection and Quantification of Transcriptome-Wide Gene Knockdown for Oligonucleotide-Based Medicines

The paper develops a dose-response model and analysis tool to accurately quantify gene knockdown effects of oligonucleotide-based medicines using next-generation sequencing data.

AttSiOff: A self-attention-based approach on siRNA de-sign with inhibition and off-target effect prediction

AttSiOff improves siRNA design by predicting inhibition and off-target effects using self-attention mechanisms, achieving better accuracy and generalization than existing methods.

Nanoparticle-mediated delivery of placental gene therapy via uterine artery catheterization in a pregnant rhesus macaque

This study delivers hIGF1-loaded nanoparticles to the placenta of a pregnant rhesus macaque via uterine artery catheterization, showing successful placental uptake and expression of hIGF1.

Lipid-Based Transfection of ZebrafishEmbryos: A Robust Protocol for NucleicAcid Delivery

The paper introduces a lipid-based transfection method for zebrafish embryos as an alternative to microinjection, improving efficiency and throughput for genetic studies.

Use of steric blocking antisense oligonucleotides for the targeted inhibition of junction containing precursor microRNAs

This study demonstrates that steric blocking antisense oligonucleotides can inhibit pre-miRNA processing by targeting structural junctions, validating their use in modulating miRNA production.

Requirements for efficient endosomal escape by designed mini-proteins

This paper identifies the requirements for efficient endosomal escape by ZF5.3, a mini-protein, showing pH-induced unfolding and lipid-BMP interaction are critical for its function.

Intranasal Delivery of shRNA to Knockdown the 5HT-2A Receptor Enhances Memory and Alleviates Anxiety

The paper demonstrates that intranasal delivery of shRNA targeting HTR2A reduces anxiety and improves memory in mice and rats.

Synthetic dosage-compensating miRNA circuits for quantitative gene therapy

A synthetic miRNA-based incoherent feed-forward loop circuit embedded in a gene delivery vector overcomes the challenge of overexpression toxicity in a mouse model of Rett syndrome gene therapy.

Non-Invasive dsRNA Delivery via Feeding for Effective Gene Silencing in Teleost Fish: A Novel Approach in the Study of Gene Function Analysis

This study demonstrates non-invasive dsRNA delivery via feeding in teleost fish to silence the dnd gene, leading to PGC development inhibition and gonadal dysplasia.

Optimized protocols for RNA interference in Macrostomum lignano

This paper optimizes RNA interference protocols in Macrostomum lignano by demonstrating that single soaking in dsRNA solution is sufficient for effective gene knockdown, reducing time and cost.

Germ granule association drives small RNA specificity for a nuclear Argonaute protein

The paper shows that HRDE-2 mediates the localization of HRDE-1 to germ granules, which is essential for its specific binding to small RNAs and proper gene silencing in C. elegans.

Cell-penetrating peptide and cationic liposomes mediated siRNA delivery to arrest growth of chronic myeloid leukemia cells in vitro

This study compares the efficiency of cell-penetrating peptides and cationic liposomes for delivering anti-BCR-ABL siRNA to inhibit CML cell growth in vitro.

Endosomolytic Peptides Enable the Cellular Delivery of Peptide Nucleic Acids

The paper demonstrates that endosomolytic peptides L17E and L17ER4 effectively deliver PNAs into human cells, enabling corrective splicing.

Genetic activation of canonical RNA interference in mice

This study shows that Dicer{Delta}HEL1/wt mice exhibit increased endogenous siRNA levels and functional RNAi in vivo, providing a new model for studying mammalian canonical RNAi.

A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system

This paper introduces a dual-targeting di-valent siRNA scaffold that can silence two genes simultaneously in the CNS of mice, demonstrating potent and programmable multi-gene modulation.

An optimized method for gene knockdown in differentiating human and mouse adipocyte cultures

This study develops an optimized siRNA transfection protocol for human and mouse adipocyte cultures throughout differentiation, improving gene knockdown efficiency.

Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors

This study evaluates tetravalent siRNA configurations for photoreceptor gene silencing, finding that tetravalent siRNA provides robust, long-lasting efficacy with a good safety profile in rodent and swine models.

AGO2 silences mobile transposons in the nucleus of quiescent cells

The paper shows AGO2 localizes to the nucleus in quiescent cells to silence mobile transposons via RNA interference.

DeepSipred: A deep-learning-based approach on siRNA inhibition prediction

DeepSipred predicts siRNA inhibition using sequence, thermodynamic, and expert knowledge features, achieving state-of-the-art performance on public datasets.

Systematic optimization of siRNA productive uptake into resting and activated T cells ex vivo

This study systematically compares siRNA delivery methods to T cells, finding lipid-conjugate-mediated delivery is most effective for both resting and activated T cells ex vivo.

Engineered dsRNA-protein nanoparticles for effective long-distance transport, delivery and gene silencing in plants

This paper demonstrates the effective long-distance transport and gene silencing in plants using cationized bovine serum albumin (cBSA) bound to double-stranded RNA nanocomplexes.

Optimization of RNAi efficiency in PVD neuron of C. elegans

The paper optimizes RNAi efficiency in C. elegans PVD neurons by expressing sid-1 under the mec-3 promoter, enhancing gene knockdown effects for dendrite development and regeneration studies.

From sequences to therapeutics: Machine learning predicts chemically modified siRNA activity

This study uses machine learning to predict the silencing activity of chemically modified siRNAs based on sequence and modification patterns, showing improved performance over existing methods.