There's a window of time in our lives we've all passed through yet still know so little about: early gestation. Researchers have found a pair of genetic deletions associated with schizophrenia that ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

In natural populations, the mechanisms of evolution do not act in isolation. This is crucially important to conservation geneticists, who grapple with the implications of these evolutionary processes ...

Published in Science, the new study reveals how a rare genetic mutation in the KEAP1 gene allows horse cells to turbocharge their energy production while also ramping up their built-in stress defenses ...

In preclinical models, Mass General Brigham researchers’ CRISPR-Cas9 gene therapy precisely repaired the genetic error that causes a rare vascular disease The ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

In a global first, UConn Health, in collaboration with Connecticut Children's, has dosed a patient in a groundbreaking clinical trial testing gene editing technology to treat glycogen storage disease ...

Metabolic dysfunction-associated steatotic liver disease (MASLD) affects nearly one in three adults around the globe. It starts when fat builds up inside the liver’s cells. Over time, this buildup can ...