A therapy that restores brain cells impaired by a rare genetic disorder may offer a strategy for treating conditions like autism, epilepsy, and schizophrenia.
NPR tells the exclusive, behind-the-scenes story of the first person with a genetic disorder to be treated in the United States with the revolutionary gene-editing technique CRISPR.
For Wilson, Tourette's syndrome means living with intrusive thoughts that flash disturbing images without warning. His novel, Nothing to See Here, was inspired by visions of spontaneous combustion.
Despite outrage over gene editing in China that affected the birth of twins, research is underway in the U.S. to assess the safety and effectiveness of CRISPR tools to edit genes in human embryos.
The market is flooded with 28 different medications for just 20,000 patients with the hereditary bleeding disorder. Yet intense competition hasn't worked to bring costs down.