The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

About 40% of East Asians suffer from alcohol intolerance, known as "Asian Flush Syndrome," caused by an ALDH2 genetic ...

A systematic review of 52 scientific papers submitted to a world-leading clinical genetics journal from multiple scientists ...

Researchers have discovered that mutations in the FOXJ3 gene act as a "master switch" failure, disrupting how the brain builds its layers and leading to FCD, a primary cause of drug-resistant epilepsy ...

dDepartment of Respiratory Medicine, National Key Clinical Specialty, Branch of National Clinical Research Center for Respiratory Disease, Xiangya Hospital, Central South University, Changsha, China ...

Mayo Clinic researchers have identified a rare mutation in the MET gene that can directly cause metabolic dysfunction-associated steatotic liver disease. The mutation disrupts the liver’s ability to ...

Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and ...

A mutation in the TDP-43 protein reduces the viability of motor neurons without triggering protein clumps, researchers found.

Some genetic mutations that are expected to completely stop a gene from working surprisingly cause only mild or even no symptoms. Researchers in ...

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and ...

Cambridge scientists have shown that when tumours first emerge, interactions with healthy cells in the underlying supportive tissue determine their ability to survive, grow, and progress to advanced ...

A team of researchers at Baylor College of Medicine and the Duncan Neurological Research Institute (Duncan NRI) at Texas ...