Researchers have developed a genome-editing strategy that targets a common cause of roughly 30 percent of rare diseases and could vastly improve access to gene-editing treatments for patients.
Stimulating the liver to produce some of the signals of the thymus can reverse age-related declines in T-cell populations and enhance response to vaccination.
Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.
The Ó³»´«Ã½ core member and gene editing pioneer describes a framework that could enable the treatment of 1,000 patients with personalized gene editing therapies by 2030.
Ó³»´«Ã½ scientists describe how data resources they helped build over more than a decade now form the foundation for cutting-edge AI and genome biology discoveries.
Large analysis of genes and proteins in lung adenocarcinoma points to possible new diagnostic and treatment approaches, and also reveals signatures of chemical exposures in patients who have never smoked.
Test of circulating tumor cells offers less invasive option for multiple myeloma surveillance, staging, and biological study compared to bone marrow biopsies
Developed in collaboration with Ó³»´«Ã½ Clinical Labs and Mass General Brigham Laboratory for Molecular Medicine, the test provides a comprehensive view of a patient’s inherited heart disease risk.
Researchers double the number of genetic factors associated with this common arrhythmia, highlighting biological pathways that could be targeted by new medicines.
Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntington’s disease and Friedreich’s ataxia.
Combining powerful imaging, perturbational screening, and machine learning, researchers uncover new human host factors that alter Ebola’s ability to infect.
Genetic datasets do not reflect the diversity of the world’s population. Alicia Martin is working to correct this through data-driven social justice.
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.