I hate to break this to you, but every child is a genetic experiment – and nature doesn’t care if things go wrong. Our genomes are awful messes created by conflicting evolutionary forces, and every ...

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the first therapy of its kind for the rare disease and making Italy’s Fondazione ...

Research links variations in the gene GRIN2A to a higher risk of developing schizophrenia and other forms of mental illness. The GRIN2A gene regulates communication between neurons by producing the ...

Scientists have identified a protein that may be key to proper sperm formation. Without it, male mice produced no viable sperm, they found. A team at the RIKEN Center for Biosystems Dynamics Research ...

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 ...

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene-writing program, inking a deal that lets it in on a prospect for alpha-1 antitrypsin deficiency (AATD) through a $150 million ...

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand ...