List view / Grid view
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.
Researchers at ETH Zurich in Switzerland have mapped the complex network cells use to repair their genetic material, revealing previously hidden vulnerabilities in cancer cells.
Rutgers researchers have discovered new insights into how polycystic kidney disease (PKD) progresses, which could lead to more targeted treatments. This breakthrough may help improve therapies for PKD patients in the future.
A team of researchers at Waseda University in Japan has identified a gene called Nwd1 that plays a key role in the development of metabolic dysfunction-associated steatohepatitis (MASH). This discovery could lead to new therapeutic strategies, potentially reshaping the treatment landscape for liver disease.
The discovery from researchers at Johns Hopkins Medicine reveals how bacteria use the CRISPR-Cas system to store viral DNA, enhancing their immunity against future infections, and potentially paving the way for new phage-based therapies
Gene silencing offers a promising approach to treating rare neurological diseases like H-ABC. With clinical trials on the horizon, find out how targeted therapies could bring real hope to patients and families.
Researchers have discovered a breakthrough method to silence MRSA's drug resistance, restoring its sensitivity to standard antibiotics and offering new hope in the fight against superbugs.
