Gene editing has moved from theory to bedside with a speed that would have seemed impossible a decade ago. A new wave of ...

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

A new genome-wide mapping method finally shows how thousands of genes connect to drive disease. Biomedical researchers are working intensively to identify the genes that contribute to disease, with ...

A new gene editor takes advantage of CRISPR-associated proteins to insert whole genes into the genome, scientists report. When you purchase through links on our site, we may earn an affiliate ...

"It's a slow-moving train wreck," Mike Graglia says about his 12-year-old son Tony's rare genetic disease with no cure. Caused by a tiny fluke of nature—a mutation in a gene known as a SYNGAP1—the non ...

Researchers have identified a potential treatment for Sandhoff and Tay-Sachs diseases -- two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and ...

Less than 30% of people with certain genetic variants go blind, despite the faulty genes previously being thought to cause ...

The Human Domainome 1—the largest library of human protein variants—reveals the cause of certain genetic disorders, paving the way for personalized medicines. “We measured every possible mutation in ...