This year marked a pivotal moment in the quest to treat Huntington’s disease, a rare but devastating form of dementia. Scientists found that an experimental gene therapy slowed the condition’s ...

KJ Muldoon is walking at home ahead of Christmas after being hospitalized last holiday due to his rare genetic disorder, CPS1 deficiency Getty KJ Muldoon, the first infant to undergo gene-editing ...

Northwestern Medicine scientists have discovered how molecular "traffic controllers" in cells influence aging and cellular senescence—a state where cells stop dividing but remain metabolically active.

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 ...

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 ...

With a new study in the journal Cell, researchers at Stanford University and Stockholm University have contributed to increased knowledge about gene regulation in human cells. How genes are turned on ...

All the cells in an organism have the exact same genetic sequence. What differs across cell types is their epigenetics—meticulously placed chemical tags that influence which genes are expressed in ...

In a step toward the wider use of gene editing, a treatment that uses Crispr successfully slashed high cholesterol levels in a small number of people. In a trial conducted by Swiss biotech company ...

What if you could flip a genetic switch to silence a gene, then turn it back on with a simple drug? For researchers, gene-switch tools offer that kind of control—and a new system called Cyclone may ...

Scientists report the first therapy to slow the deadly brain disease. A one-time gene therapy treatment has shown remarkable success in slowing the progression of Huntington's disease in a preliminary ...