GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

No gene acts alone: interacting variants and protein partnerships can worsen, mask or even rescue disease risk, demanding ...

The new molecules selectively reduced inflammatory signaling in human immune cells without affecting other immune pathways. Treatment with the molecules also reduced inflammation in mice with the ...

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 ...

A nationwide Australian pilot screened more than 10,000 adults aged 18–40 years for high-risk genetic variants linked to ...

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 ...

Huntington's disease is caused by an inherited genetic change that causes production of an altered protein. This leads to nerve damage in areas of the brain that control movement and thinking.

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 ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

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 ...

Scientists at the Icahn School of Medicine at Mount Sinai and collaborators have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.