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.
When people think about prion mutations, their mind jumps to things that cause disease. But as a Ó³»´«Ã½ researcher points out, some prion mutations can protect against disease, and point to potentially valuable research avenues.
Researchers deliver CRISPR-Cas9 directly into the inner ear hair cells of mice, preventing hearing loss in animal model of genetic progressive deafness.
Similar mutations in the gene SPOP have completely opposite effects in prostate versus endometrial cancers. What does that mean for efforts aimed at functionally interpreting cancer genetic findings?
Inspired by rare genetic disease, researchers develop a compound that can protect kidney cells from death and restore kidney function in multiple animal models of progressive kidney disease.