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 ӳý 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.
ӳý 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 ӳý 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.
Bill Sellers, head of ӳý’s Cancer Program, talks about how to discover new classes of genetic dependencies that could move therapies closer to curing cancer patients.
Researchers at ӳý and MIT report the first injectable “priming agents” for liquid biopsy, which increased circulating tumor DNA by more than 10-fold in mice.
By adapting virus-like particles to carry the machinery for a type of gene editing called prime editing, scientists have corrected disease-causing mutations in animals and increased editing efficiency.