A deep-learning model trained on human data reveals that promoter mutations may explain a significant portion of unsolved ...

Recent findings shed new light on the critical role of TET1, a pivotal player in epigenetic regulation, in the development ...

The Rubin telescope, sitting at the top of the Cerro Pachon mountains in Chile, captured galaxies 55 million lightyears away. Thomas Mulligan weighs the promise and ethical risks of gene editing to ...

Genetically engineered skin grafts exhibited a long-term ability to heal large, chronic wounds in patients with recessive ...

Repositioning genes awakens fetal hemoglobin to treat disease. CRISPR editing may change future gene therapy. Researchers ...

Using gene editing in a preclinical model, researchers at UT Southwestern Medical Center blocked the symptoms of a rare smooth muscle disease before they developed. Their findings, published in ...

Moving toward clinical development, the researchers demonstrated efficient delivery of mRNA mitochondrial base editors using lipid nanoparticles.

Huntington’s disease is a rare genetic disorder affecting movement, memory, and mood. Here’s what we know—and what’s ahead.

The newly launched South African Blood Regulatory (SABR) dataset reveals how genetic variation influences blood traits and ...

Most gene therapies being studied for eye diseases are in earlier phases of development, and a third of the gene therapies ...

Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C.

Scientists have identified human genes that act like light switches, flipping on or off and influencing fertility and disease risk.