Regeneron Pharmaceuticals has demonstrated that its gene therapy, DB-OTO, can restore hearing in children born with profound deafness due to OTOF gene mutations. The therapy uses a dual-vector viral system…
Researchers at Mass General Brigham have identified a rare genetic variant in the RELN gene that protected a man from Alzheimer's symptoms for nearly three decades. This discovery highlights a…
A global research team has identified "Obelisks," a new class of viroid-like genetic entities found in the human gut and mouth. These circular RNA structures are distinct from known viruses…
Researchers at the National Institutes of Health identified a genetic disorder called DADA2 that causes early-onset strokes and chronic inflammation. By pinpointing mutations in the ADA2 gene, the team developed…
Researchers at Mount Sinai have discovered that spaceflight can lead to somatic mutations in the blood-forming stem cells of astronauts. These mutations, known as clonal hematopoiesis, were found at higher-than-expected…
An international research consortium has integrated data from nearly 50 studies to create the Human Lung Cell Atlas, the most detailed map of the respiratory system ever produced. This open-access…
Researchers at the National Institutes of Health have identified a new, life-threatening autoinflammatory condition called VEXAS syndrome. Caused by somatic mutations in the UBA1 gene, the discovery marks a shift…
