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Insilico Medicine and Ribo enter strategic collaboration agreement: Leveraging AI platform and automated lab for end-to-end empowerment of RNA interference and oligonucleotide therapeutics development

Insilico Medicine's Pharma.AI platform combines with Ribo's expertise in oligonucleotide therapeutics, aiming to boost efficiency and certainty of clinical research. The partnership enables end-to-end empowerment of RNA interference and oligonucleotide therapeutics development.

Protecting crops: Researchers open up new avenue to combat a widespread plant virus

Researchers at Martin-Luther-Universität Halle-Wittenberg have developed a new avenue to combat the Cucumber mosaic virus by directing the plant's natural defences. The RNA-based active agents have shown high efficacy in laboratory experiments, protecting 80-100% of treated plants from infection.

SourceMartin-Luther-Universität Halle-Wittenberg·JournalNucleic Acids Research·TypeExperimental study·DateMar 18, 2025

siRNA-AGO2 complex inhibits bacterial gene translation: a novel therapeutic strategy for superbug infection

A novel therapeutic strategy for superbug infections has been discovered using the siRNA-AGO2 complex. This complex inhibits bacterial gene translation by delivering AGO2-loaded siRNA into bacterial cytoplasm, downregulating resistance genes and converting multidrug-resistant bacteria into methicillin-sensitive ones.

SourceNanjing University School of Life Sciences·JournalCell Reports Medicine·TypeExperimental study·DateMar 6, 2025

Rice-BCM research achieves gene-editing breakthrough that could improve treatment for liver disease, other disorders

The Rice University lab, in collaboration with Baylor College of Medicine, has developed a new gene-editing strategy called Repair Drive that improves the effectiveness of gene therapies in the liver. The technique enables the repair of liver cells at higher rates and equips them with a selective advantage to outcompete incorrectly edi...

SourceRice University·JournalScience Translational Medicine·TypeExperimental study·DateFeb 13, 2025

Research Spotlight: Lipid nanoparticle therapy developed to stop tumor growth and restore tumor suppression

Researchers developed a novel approach using lipid nanoparticles to deliver mRNA and siRNA, restoring tumor suppressors and inhibiting tumor drivers in prostate cancer cells. This technique holds promise for treating various types of cancer by targeting specific pathways related to tumor growth and suppression.

SourceMass General Brigham·JournalACS Nanoscience Au·TypeExperimental study·DateJan 9, 2025

Unlocking RNA’s benefits to combat complex diseases

Researchers at the University of Ottawa have developed a nanoparticle strategy to deliver both mRNA and siRNA, enhancing and interfering with multiple gene and protein expressions. This approach holds significant promise for treating major diseases like cancer and cardiovascular diseases.

SourceUniversity of Ottawa·JournalNanoscience·TypeExperimental study·DateDec 9, 2024

Solving the side effect problem of siRNA drugs for genetic disease treatment using formamide

Researchers at Nagoya University have developed a method to chemically alter siRNAs, reducing off-target effects and improving the safety of siRNA drugs for genetic therapy. By modifying the seed region of siRNAs with formamide, they achieved suppression of off-target effects with higher efficiency than existing chemical modifications.

SourceNagoya University·JournalNucleic Acids Research·DateSep 6, 2024

Effectiveness of using siRNA to treat Huntington’s disease

A new study published in Nucleic Acid Therapeutics found that siRNA reduces huntingtin mRNA levels in the cytoplasm but not in the nucleus of mouse brains, suggesting a limitation in its effectiveness for treating Huntington's disease. The research highlights the importance of understanding the structure and function of nuclear RNA to ...

SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalNucleic Acid Therapeutics·TypeExperimental study·DateJul 22, 2024

RNA inhibitor is shown safe and effective in reducing a wide range of cholesterol and triglyceride levels in the blood in Mount Sinai-led clinical trial

A clinical trial led by Mount Sinai researchers shows zodasiran, an RNA inhibitor targeting ANGPTL3, significantly reduces various types of cholesterol and triglycerides. The therapy also lowers apolipoprotein B, a lipid-transporting protein linked to heart disease.

SourceThe Mount Sinai Hospital / Mount Sinai School of Medicine·JournalNew England Journal of Medicine·TypeRandomized controlled/clinical trial·DateMay 29, 2024

Scientists from IOCB Prague are expanding the possibilities of using RNA in gene medicine

Scientists from IOCB Prague have created a novel composite vector nanomaterial for transporting ribonucleic acid (RNA) into cells, ensuring its non-toxicity. This breakthrough aims to overcome the obstacle of nucleic acid vectors' toxicity and pave the way for gene therapy applications.

SourceInstitute of Organic Chemistry and Biochemistry of the Czech Academy of Sciences (IOCB Prague)·JournalAdvanced Functional Materials·TypeExperimental study·DateMay 15, 2024

Evaluation of on- and off-target effects of self-assembled EGFR siRNA delivery system

The research group evaluated the on- and off-target effects of a self-assembled EGFR siRNA delivery system, finding potent interference with only 1/2000000 of synthetic transfection amount. Transcriptomic analysis revealed minimal off-target effects in normal tissues and significant anti-EGFR effects in tumor cells.

SourceNanjing University School of Life Sciences·JournalClinical and Translational Medicine·DateFeb 6, 2024

An ancient anti-cancer mechanism: DISE

Researchers at Northwestern University identified a new evolutionarily conserved RNAi-based form of cell death called Death Induced by Survival gene Elimination (DISE), which targets essential survival genes in cancer cells. This mechanism is ancient and effective against all cancers tested.

SourceImpact Journals LLC·JournalOncotarget·TypeCommentary/editorial·DateOct 3, 2023

RNA nanoparticle therapy stops the spread of incurable bone marrow cancer

Researchers have created an RNA nanoparticle therapy that disables the pathways through which multiple myeloma cells travel, stopping their spread. The therapy targets the microenvironment of the cancer and prevents the production of a protein that attracts cancer cells to blood vessels.

SourceUniversity of Pennsylvania School of Engineering and Applied Science·JournalProceedings of the National Academy of Sciences·DateJun 13, 2023

Mass Eye and Ear Hearing scientists use drug-like cocktail to regenerate hair cells in preclinical study

Researchers have created a drug-like cocktail that successfully regenerated hair cells in a mouse model by reprogramming genetic pathways within the inner ear. The approach could lead to clinical trials for a gene therapy that can be administered to people with hearing loss, potentially treating up to 90% of cases.

SourceMass Eye and Ear·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateApr 18, 2023

New strategy enables targeted treatment of rheumatoid arthritis

A new strategy for treating rheumatoid arthritis has been proposed, integrating small interfering RNAs and Prussian blue nanoparticles to silence proinflammatory cytokines and scavenge reactive oxygen species. The approach was tested in a mouse model, showing improved therapeutic efficacy and real-time monitoring capabilities.

SourceChinese Academy of Sciences Headquarters·JournalProceedings of the National Academy of Sciences·TypeMeta-analysis·DateOct 21, 2022

In vivo self-assembled siRNA as a modality for combination therapy of ulcerative colitis

A new study developed a synthetic biology strategy for delivering siRNAs through small extracellular vesicles, alleviating intestinal inflammation and exerting synergistic effects against ulcerative colitis. The approach offers an attractive option for combination therapy and holds promise for treating autoimmune diseases.

SourceNanjing University School of Life Sciences·JournalNature Communications·TypeExperimental study·DateSep 29, 2022

Polymersomes efficiently deliver siRNA to treat breast cancers in preclinical model

Researchers have developed biodegradable nanovesicles that efficiently encapsulate and deliver PARP1 siRNA to breast cancer tumors in mice, inhibiting oncogene expression and extending survival. The polymersomes, assembled from three biodegradable block copolymers, have strong potential for precision-targeted therapeutic carriers.

SourceUniversity of Alabama at Birmingham·JournalACS Applied Bio Materials·TypeExperimental study·DateMay 24, 2022

Novel supramolecular CRISPR–Cas9 carrier enables more efficient genome editing

A team of researchers from Kumamoto University has developed a transformable polyrotaxane carrier that can facilitate genome editing using Cas9RNP with high efficiency. The carrier, called amino-PRX, is multi-step transformable and has low cytotoxicity, making it an enormously promising candidate for safe and efficient delivery.

SourceKumamoto University·JournalApplied Materials Today·TypeExperimental study·DateMay 11, 2022

Cleveland Clinic-led trial finds that experimental ‘gene silencing’ therapy reduces lipoprotein(a), an important risk factor of heart disease, by up to 98%

A Cleveland Clinic-led trial found that an experimental 'gene silencing' therapy significantly reduced blood levels of lipoprotein(a), a key driver of heart disease risk, by up to 96%-98%. The therapy targets the gene responsible for Lp(a) production and has shown strong efficacy with no major safety concerns reported.

SourceCleveland Clinic·JournalJournal of the American Medical Association·DateApr 3, 2022

How do mobile siRNAs mediate transgressive methylation in grapevine plants?

Recent research reveals that mobile siRNAs play a crucial role in transgressive methylation in grapevine plants, influencing phenotypic changes in heterografts. The study demonstrates bi-directional small RNA transfers between graft partners, with preferential transfer of scion-derived smRNAs to the rootstock.

SourceNanjing Agricultural University The Academy of Science·JournalHorticulture Research·TypeNews article·DateMar 29, 2022

Discovery of a universal system for transporting nucleic acids into cells

A team of researchers from IOCB Prague has discovered a new type of nanoparticles capable of safely transporting various types of nucleic acids used for therapeutic purposes into cells. The universal nature of their system sets it apart from existing solutions, allowing for efficient transport of mRNA and other RNA molecules into cells.