Scientists at Scripps Research have pinpointed dozens of human proteins that SARS-CoV-2 needs to complete its full life cycle. Two promising drug targets, perlecan and BIRC2, show potential in blocking coronaviruses at multiple stages of infection.
A recent study identifies LPCAT1 as a critical culprit in TNBC's aggressive behavior, which can be silenced with siRNA-delivering nanoparticles. This approach starves tumors of their energy supply, dramatically reducing tumor growth and lung metastasis.
The single-celled parasite Entamoeba histolytica infects 50 million people each year and can kill nearly 70,000. Researchers have discovered that it damages tissue through a process called trogocytosis, where it takes bites out of human cells and ingests their fragments to evade the immune system.
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.
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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.
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...
Researchers identified peri-centrosome-localized siRNAs in C. elegans, which accumulate at the centrosome during cell cycle and are essential for development. The study provides new insights into RNAi-mediated gene regulation in C. elegans.
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.
Researchers at Memorial Sloan Kettering Cancer Center discovered an unrecognized role for ALAS1 in regulating microRNAs, which could improve the efficacy of siRNA drugs against disease-causing genes. The finding offers new potential for developing targeted therapies against cancer and other diseases.
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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.
The CTPA/siCSF1R system enhances the tumor microenvironment and supports precise photoimmunotherapy by delivering siRNA to tumor-associated macrophages (TAMs). This innovative approach reprograms TAMs to promote a strong immune response against tumors.
A new study reveals that two specific genes in the RNA interference pathway play a crucial role in preventing virus transmission from parent to progeny in plants. This discovery could lead to healthier crops and potentially reduce the transmission of diseases like Zika from mothers to human children.
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.
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Researchers have developed two new methods to produce circular RNAs, which can silence genes and serve as templates for making therapeutic proteins. These circular RNAs display enhanced stability and biological activity in heart muscle cells and neurons.
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 ...
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.
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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.
Researchers at UC Riverside demonstrate a new vaccine strategy targeting a common viral genome part, eliminating the need for annual booster shots. The vaccine uses small RNA molecules to boost the immune system, making it safe for babies and those with weakened immunity.
Rockefeller University researchers have discovered a new way to target the oncogene behind a rare and often deadly liver disease using small interfering RNAs (siRNAs). The approach involves delivering siRNAs inside fibrolamellar hepatocellular carcinoma (FLC) cells through a surface receptor, blocking the oncogene from producing illnes...
A new approach to fighting HIV has been developed using RNA, specifically small interfering RNAs (siRNA), which regulate gene expression in cells. This nanomedicine was shown to reduce HIV replication by 73% and is intended for vaginal application to prevent sexual transmission.
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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.
Researchers investigated role of estrogen receptors in lateral septum regulating social anxiety in male mice, finding that ERβ plays a crucial role in neural circuitry. Knockdown effects on ERβ gene expression showed increased social anxiety, highlighting its importance in social situations.
A novel synthesis method enables easy linkage of therapeutic oligonucleotides to peptide markers, streamlining the process and making it more accessible and cost-effective. This breakthrough has the potential to produce more effective and targeted RNA-based drugs.
Researchers developed novel nanoparticles that deliver RNA to microglia immune cells, reducing inflammation linked to Alzheimer's disease. The study showed a 42% reduction of PU-1 expression and multiple inflammatory markers in human cell cultures and mice models.
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Lepodisiran, a short interfering RNA, targets hepatic synthesis of apolipoprotein(a), reducing serum lipoprotein(a) concentrations. Phase 1 study results support further investigation of lepodisiran's efficacy in treating elevated lipoprotein(a) levels.
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.
A team of Chinese and UK researchers has identified superoxide dismutase 1 (SOD1) as a potential target for reversing drug resistance in ovarian cancer. By using nanoparticles to deliver siRNA that reduces SOD1 levels, the study showed reduced growth and decreased resistance to cisplatin in female mice.
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.
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A new research centre will focus on developing new types of RNA medicine for treating metabolic diseases. The centre, led by Professor Jørgen Kjems at Aarhus University, aims to create targeted treatments for conditions like diabetes and atherosclerosis.
Researchers successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. The technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses.
Researchers investigated the roles of STAT3α and STAT3β in aggressive breast cancer and found that differential silencing of these isoforms leads to changes in STAT3 activation. This study emphasizes the importance of distinguishing between STAT3 isoforms for accurate cancer diagnosis and therapy.
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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.
Researchers at University of Pittsburgh have designed novel nanoparticles that co-deliver a chemotherapy drug and a novel immunotherapy, shrinking tumors in mouse models of colon and pancreatic cancer. The therapy silences a gene involved in immunosuppression by blocking Xkr8 protein distribution on the cell membrane.
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.
Researchers at Harvard's Wyss Institute discover new class of immunostimulatory dsRNAs that potently induce IFN-I production while limiting inflammation. The dsRNAs inhibit pandemic viruses, including SARS-CoV-2, in mouse and human Organ Chip models.
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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.
Researchers developed nanoparticles to deliver anti-inflammatory payloads directly to inflamed blood vessels, significantly increasing survival chances and delaying rupture onset. Targeting specific NF-kappaB subunits improved therapeutic effects with fewer adverse effects.
A team of scientists has discovered the genetic mechanism behind the emergence of highly resistant fungal strains, such as Cryptococcus neoformans. The researchers found that transposon mobility is controlled by small interfering RNA (siRNA), and that disabling siRNA can lead to resistance.
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.
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Researchers are developing techniques to use milk-exosomes to deliver siRNAs to brain tumors, aiming to reduce tumor growth and improve patient outcomes. The goal is to develop large-scale production methods and eventually genetically modify cows to produce therapeutic exosomes.
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.
Researchers developed a virus that infects cancer cells, killing them while sending signals to nearby uninfected cells for viral attack. This approach shrinks tumors and enhances cancer-killing efficacy in various models, including pancreatic and ovarian cancers.
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Scientists create nanoparticles coated in mannose to block production of scar-promoting protein in lung cells. The treatment holds promise for preventing severe lung scarring disease.
Researchers found that SLN360, an experimental siRNA therapy, significantly reduced blood levels of lipoprotein(a), a lesser-known driver of heart disease risk. Participants who received higher doses saw their lipoprotein(a) levels drop by up to 98% and remained lower even after five months.
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.
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.
A research team at TUM has successfully used specific enzymes to destroy the genetic information of SARS-CoV-2 directly after it penetrates the cell. The study found that targeting the viral genome with siRNAs is most effective when the virus has just penetrated into the cell.
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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.
Researchers at SMU found that decreasing TIGAR protein levels in cervical cancer cells makes them more responsive to chemotherapy drugs at low doses. This discovery offers a potential new treatment approach for HPV-induced cervical cancer with fewer side effects.
Researchers have created nanoparticles that can effectively deliver and protect siRNA, a promising class of therapeutics targeting harmful genes. This breakthrough aims to improve the treatment of diseases by fine-tuning mRNA production, offering a new approach to managing irregular protein expression.
Researchers at Einstein College of Medicine developed a topical drug that regenerates and restores function of erectile nerves damaged by radical prostatectomy. The siRNA gel enhanced nerve regeneration and restored nerve function in rat models, restoring erectile function.
A new siRNA delivery system allows for in vivo self-assembled small RNA synthesis, enabling potent target gene silencing in various diseases. This strategy overcomes the limitations of conventional delivery methods, offering a promising approach to RNAi therapy.
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Scientists develop patented RNA-based therapy to degrade overexpressed PMP22 protein, restoring nerve function and strength in mice. The treatment uses biocompatible siRNA nanoparticles and controls particle size and injection amount.
A novel nanoparticle platform facilitates therapeutically effective delivery of encapsulated agents to the brain, opening possibilities for treating neurological disorders. The technology showed three times more accumulation in brain than conventional methods and was therapeutically effective in mouse models.
Georgetown University researchers successfully used RNA molecules to suppress SARS-CoV-2 protein production, potentially leading to an inhalable drug that mitigates viral chaos. The approach targets viral machinery within cells via siRNA, degrading messenger RNA and reducing infection spread.
Researchers develop new strategy for stabilizing siRNAs using ODAGal4, which improves biological and thermal stability without compromising gene-silencing activity. This approach has great potential for siRNA-based therapies and reduces cytotoxicity.
A recent study published in Nature Plants reveals that short double-stranded small interfering RNAs (siRNAs) are the primary messengers responsible for RNA interference in plants. These siRNAs can travel vast distances, enabling plants to modulate gene expression at a distance and adapt to their environment through phenotypic plasticity.
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Researchers develop siRNA-based treatment for psoriasis using ionic-liquid technology, reducing inflammation and symptoms without systemic side effects. The delivery method opens up new possibilities for treating debilitating dermatological disorders.
Small interfering RNA (siRNA) shows potential for treating human diseases by selectively silencing pathological pathways. The review examines siRNA therapeutics in preclinical and clinical trials, challenges faced in siRNA therapy, and the feasibility of combining siRNA with anticancer drugs.
Researchers have discovered a new target and therapy to fight NAFLD, a major health issue affecting 25% of the world population. Using siRNA to silence MCJ protein, they found reduced liver steatosis and fibrosis in preclinical mouse models.
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