FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

For genetic conditions like Duchenne muscular dystrophy, there is little doctors can do to slow or treat the condition other than trying to manage symptoms, since only addressing the genetic changes responsible can help halt the disease. Earlier today (June 22), the U.S. Food and Drug Administration (FDA) approved the first such intervention, a gene … Read more

Scientists Have Built a Human ‘Pangenome’ to Better Reflect Diversity

For two decades, scientists have been comparing every person’s full set of DNA they study to a template that relies mostly on genetic material from one man affectionately known as “the guy from Buffalo.” But they’ve long known that this template for comparison, or “reference genome,” has serious limits because it doesn’t reflect the spectrum … Read more

How Jennifer Doudna’s Life Has Changed Since Discovering CRISPR 10 Years Ago

Jennifer Doudna was staring at a computer screen filled with a string of As, Cs, Ts, and Gs—the letters that make up human DNA—and witnessing a debilitating genetic disease being cured right before her eyes. Just a year earlier, in 2012, she and microbiologist Emmanuelle Charpentier had published a landmark paper describing CRISPR-Cas9, a molecular … Read more