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Scientists have used fruit flies to study 100 genes linked to Alzheimer’s disease and how these genes affect brain structure, function and stress resilience. The research could help guide new treatments in the future.
Scientists have linked rare mutations in SPNS1 to a previously unknown lipid recycling pathway in lysosomes, revealing how faulty fat processing can trigger muscle and liver disease.
Researchers have refined a cutting-edge DNA sequencing tool that reveals how mutations accumulate in healthy tissues as we age, offering insights into the earliest stages of cancer development.
Northwestern University researchers have created DNA-coated nanoparticles that deliver CRISPR into cells three times more effectively while reducing toxicity. The advance could overcome one of the biggest barriers to gene-editing therapies.
An existing asthma drug has been shown to almost completely prevent life-threatening allergic reactions to food in mice, after researchers found a previously unknown genetic pathway linked to anaphylaxis.
Can a cholesterol enzyme help treat an untreatable liver disease? Esperion’s ACLY programme is using multiomic and preclinical data to evaluate its potential in primary sclerosing cholangitis.
Single-cell and spatial technologies are giving researchers an unprecedented view of how brain diseases like Alzheimer’s really work. The result? Faster discovery, clearer targets and a new path towards more effective treatments.
Researchers at the University of Basel and Roche have discovered that slowing the intracellular transport of RNA-based drugs significantly improves their therapeutic impact.
Researchers at Tel Aviv University have developed a new mouse model that accurately mimics a rare and severe genetic disorder caused by mutations in the GRIN2D gene – allowing for study of the disease’s progression and the testing of potential drug therapies.
Researchers have developed a novel gene therapy approach that reactivates dormant genes by repositioning them closer to genetic switches called enhancers - showing promise for treating blood disorders like sickle cell disease.
Researchers at the Fralin Biomedical Research Institute are developing a targeted KCNT1 inhibitor that has shown early promise in reducing seizures in preclinical models.
Alltrna is redefining the future of rare disease treatment with engineered tRNA therapeutics that target genetic mutations - not just individual diseases. CEO Michelle Werner shares how this bold, mutation-driven approach could unlock universal treatments for thousands of patients long overlooked by traditional medicine.
Tyra Biosciences has announced new data showing that its investigational therapy, TYRA-300, improves bone growth and corrects skeletal abnormalities in preclinical models of achondroplasia and hypochondroplasia.
Huntington's disease remains a major challenge, but allele-selective gene editing offers new hope. By targeting only the mutant gene, this approach could provide a one-time, durable treatment. Life Edit’s Dr Amy Pooler reveals how this innovative therapy could reshape HD treatment.
Researchers at Keio University have developed long-lasting, functional human liver organoids from frozen hepatocytes, marking a huge moment in organoid science.
