In the 1990s, Lee Sweeney, a physiologist at the University of Pennsylvania, demonstrated that inserting the gene encoding insulin-like growth factor 1 (IGF-1) into the muscle cells of mice increased ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases. KJ Muldoon was born with a rare genetic disorder, ...

Earth's biodiversity is in crisis. An imminent "sixth mass extinction" threatens beloved and important wildlife. It also threatens to reduce the amount of genetic diversity—or variation—within species ...