Researchers discovered that brain enzyme OTULIN regulates tau protein accumulation and has implications for treating neurodegenerative diseases. The study revealed OTULIN's role in controlling gene expression and RNA metabolism, suggesting a potential therapeutic target.
Researchers have developed a system to selectively switch off the key molecule of NMD, allowing them to observe its function in human cells with unprecedented precision. The study reveals that NMD not only prevents errors but also acts as an important regulator of gene activity.
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Researchers developed a modified sugar that increases the effectiveness and safety of antisense oligonucleotides, a treatment strategy for central nervous system disease. The modification, called BNAP-AEO, decreases toxic side effects while improving gene silencing in brain cancer cells and mice.
Scientists have discovered an additional source of genetic mutations that cause rare conditions like Huntington's disease. Expanded CAG repeat RNA can form aggregates that reduce global protein synthesis and lead to neurotoxicity.
A team of scientists has identified a key player in the coupling between early transcription termination and RNA degradation. The ARS2 protein recruits ZC3H4, which interacts with the NEXT complex to target nascent transcripts for degradation.
Researchers developed a strategy targeting RNA recycling enzymes to prevent harm from cancer genes MYC, JUN and MIR155. This approach showed promise in slowing tumor growth in cancer patients.
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Researchers designed high-fidelity Cas13 variants using mutagenesis to reduce collateral effects, demonstrating their feasibility and efficiency in mammalian cells and animals. The most promising variant, hfCas13d, showed marked reduction in off-target genes and no detectable collateral damage, supporting its potential for gene therapy.
A new mechanism has been discovered that decorates the end tails of RNA molecules in a parasite causing sleeping sickness, preventing their degradation and potentially increasing virulence. This fundamental discovery opens new avenues for treatment strategies for this disease, as well as other RNA-based infections/diseases.
McGill researchers uncover a cellular mechanism contributing to communication breakdown between neurons in Alzheimer's disease. They found inadequate levels of the protein RBFOX1, which stabilizes RNAs involved in synaptic transmission, may be a factor in faulty connections characteristic of Alzheimer's.