Tiffany Fransen's lifelong clumsiness turned out to be a rare neurodegenerative disease. Friedreich’s ataxia leads to muscle ...

In an exciting scientific first, researchers at the Allen Institute successfully designed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders (SRD) in mice. These are a ...

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

Affecting 1 in 500 people, hypertrophic cardiomyopathy is a condition in which the walls of the left ventricle, the heart's main pumping chamber, become abnormally thick. "HCM is one of the primary ...

Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only minor ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ...

Please provide your email address to receive an email when new articles are posted on . The expression of GSDMB was high in children with a 17q21 genetic variant. These children did not have ...

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

The Iowa State University Extension and Outreach Dairy Team monthly webinar series continues from noon to 1 p.m. April 2. The program will help Holstein breeders understand the muscle weakness genetic ...

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

A 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment was released ...