For decades, the dream of fixing harmful mutations in mitochondrial DNA felt out of reach. Scientists have long known these ...

A progressive neurological disorder affecting 10 million people globally, Parkinson’s can cause disruptive symptoms such as ...

Logan Cyr, 16, joined a global accelerator program for teens that enables him to research cures for genetic malformations.

DNA makes RNA, RNA makes protein, protein makes phenotype—was the guiding framework for understanding inheritance and disease ...

Autism has at least four subtypes, an analysis of more than 5,000 children’s genes, traits and developmental trajectories has ...

President Trump's "Big Beautiful' Bill" includes a provision incentivizing the study of "orphan drugs" to treat rare diseases.

Scientists discovered a blood stem cell mutation that may lower Alzheimer’s risk. Learn how this breakthrough could shape ...

Picture juicy red tomatoes on the vine. What do you see? Some tomato varieties have straight vines. Others are branched. The ...

The research is a transformative step toward understanding the condition's genetic underpinnings and potential for ...