A study led by Aaron Hobbs, Ph.D., and Rachel Burge, Ph.D., at MUSC Hollings Cancer Center, reveals why a specific gene ...
Experiments on an ultra-rare genetic mutation that causes neurodegeneration in children have helped uncover a new mechanism by which brain cells die. A team led by scientists at the German research ...
A vanishingly rare genetic glitch in a single enzyme can erase a newborn’s brain cells in a matter of weeks, leaving doctors with almost no way to intervene. Researchers have now traced that ...
Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...
Ultra-rare disorders are those affecting fewer than one person in 50,000. Sometimes, indeed, they are unique. And unique ...
No gene acts alone: interacting variants and protein partnerships can worsen, mask or even rescue disease risk, demanding ...
A rare form of diabetes affecting newborn babies and caused by a genetic disorder has been discovered by scientists in the UK ...
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...
Aurora Therapeutics today announced its official launch to transform personalized gene editing from a one-patient breakthrough into a scalable model capable of bringing therapies to millions of ...
It began quietly, tucked away in the immune systems of a few dozen people scattered across the globe. They weren't superheroes. They didn't wear capes. But they carried something extraordinary, an ...