A new biotechnical vector, VIBV, combines viral mimicry with synthetic nanotechnology to deliver targeted RNA therapies for cancer treatment. The vector uses a spindle-shaped nanostructure and polyethylene glycolylated liposomal coat to evade immunity and extend circulation.
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.
Researchers have discovered that targeting a specific mutation in fibrolamellar tumors can reduce tumor growth in mice, offering a promising approach to treating this nearly incurable cancer. The findings highlight the potential for novel therapies against an intractable disease.
Researchers from HKUST have identified secondary RNA elements that control DICER's cleavage activity, improving miRNA expression and shRNA design for effective gene silencing. The study provides a foundation for designing accurate and efficient short-hairpin RNAs.
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Researchers at Toyohashi University of Technology have developed DNA stamper injections using nanoscale-tipped wire arrays to deliver biomolecules into live neuronal cells within brain tissues ex vivo and in vivo. This technique allows for the efficient genetic modification of brain cells, making it a powerful tool for neuronal research.
Researchers used a genetic screen to identify genes essential for neuron survival, including those involved in cellular metabolism. The study also uncovered new targets for treating Huntington's disease, such as the Nme gene family.
Researchers investigated shRNA therapy for Huntington's Disease, identifying novel methods to modulate construct expression and reduce off-target effects. The study proposes two feedback mechanisms to control shRNA expression, inspired by synthetic biology.
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Scientists at CSHL have devised an algorithm that improves RNA interference technology harnessing short hairpin RNAs (shRNAs) for effective gene knockdown. The new algorithm, called shERWOOD, was trained on a massive parallel assessment of shRNA potency and can predict the efficacy of new sequences.
Researchers at Mirimus Inc. developed a new technology to enhance RNA interference efficiency and accuracy, enabling functional gene annotation in normal homeostasis and disease. The new approach uses an optimized microRNA backbone to increase the success rate of RNAi screens and models.
A recent study found that targeting the JAK1 and JAK2 tyrosine kinase pathways can increase tumor cell susceptibility to natural killer cell-mediated death. Pharmacological inhibition of these pathways was shown to enhance tumor cell killing, making them a promising target for cancer therapy.
Researchers developed a novel bipartite gene therapy approach to temporarily preserve photoreceptors in a mouse model of retinitis pigmentosa. The treatment targets defective phosphodiesterase metabolism, reducing cGMP and Ca2+ levels, and showing promise for treating this genetic disorder.
A CSHL-led team developed a powerful method to identify potent RNAi triggers, allowing biologists to fully exploit this natural mechanism. The approach revealed new insights into small RNA biogenesis and improved the recipe for creating potent RNAi triggers.
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Research on heparanase-specific shRNA reveals its potential as a novel therapeutic strategy for human gastric cancer. The study successfully knocked down HPA expression in gastric cancer cells, leading to decreased invasiveness and metastasis. This finding suggests a new approach for treating cancers overexpressing HPA.
Researchers have identified a host of genes that cancer cells depend on for survival, including serine/threonine kinase 33 and polo-like kinase 1. Targeting these kinases could potentially lead to effective treatments for various types of cancer.
Researchers have developed a method to quickly identify shRNA that turns genes on and off, enabling complex genetic screens at minimal cost. The tool has the potential to revolutionize the study of gene function in mammals, paving the way for targeted therapeutics.
Researchers from MIT and Alnylam Pharmaceuticals have shown that siRNA does not interfere with the microRNA pathway, achieving 80% silencing of target genes in mice and hamster liver cells. This approach could lead to treatments for a wide range of diseases.
A human genome-wide RNAi library has been developed by Cold Spring Harbor Laboratory, enabling companies to identify and validate target genes for new drugs. The library targets over 10,000 human genes with sequence-validated short hairpin RNA molecules.
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Researchers have created new RNA libraries that can selectively inactivate human genes, enabling efficient screening for genetic defects. The libraries, made widely available to the research community, will greatly aid in understanding human biology and disease.