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

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

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 ...

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

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

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

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

Gene therapy advancements necessitate improved delivery infrastructure and comprehensive data collection across age groups to address logistical complexities and policy gaps. Multidisciplinary ...

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

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