A one-time genome-editing treatment restores motor function and extends lifespan in an animal model of a neuromuscular disease that is the leading genetic cause of infant mortality.
Scientists have solved the structure of an important complex of RAS-pathway proteins, explaining how known mutations lead to disease and suggesting potential new binding sites for cancer drugs.
By studying individual cardiac cells from heart failure patients, researchers have identified molecular signatures that point to biological mechanisms of disease.
International collaborations analyze common and rare DNA variants in hundreds of thousands of people, further elucidating genetic roots of psychiatric disorder
Researchers will analyze the DNA of tens of thousands of people from five countries in East and South Asia to find genetic markers for the psychiatric condition.
A study of respiratory syncytial virus genomes reveals that multiple lineages were circulating within the United States last fall, rather than one highly transmissible variant.
Broad researchers discover that changes in immune responses and the gut microbial community could underlie repeated occurrences of these common infections.
By introducing cancer-causing mutations into healthy skin cells step-by-step, Broad scientists have created models of skin cancer that can reveal the effects of mutations.
By using genetic data on multiple traits from people of non-European ancestry, scientists have improved the accuracy of polygenic scores in predicting disease risk for all.
The $400,000 award recognizes the far-reaching medical impact of Dr. Caruthers’ development, in the early 1980s, of an efficient and fast method to synthesize nucleic acids.