For a technology that costs millions, a collaborative approach may improve efficiency and allow the science to live up to its ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

For decades, geneticists have known that most common illnesses are not caused by a single rogue gene but by intricate ...

An Illinois family pushed for both boys to receive the potentially life changing medical treatment after one was approved and ...

Amid news of the splashy $4.8 billion Amicus buy, BioMarin has slipped in the discontinuation of a genetic liver disease ...

A new genetic mapping strategy reveals how entire networks of genes work together to cause disease, filling in the missing ...

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

The study focused on inherited retinal degenerations (IRDs), a group of genetic diseases that lead to progressive vision loss ...

Sometimes, a small error in the sequence of DNA can lead to a very serious disease. Scientists have identified many mutations ...

A new study challenges what's long been assumed about genetic variants thought to always cause inherited blindness.