This new therapy involves an innovative two-step strategy: Precision gene delivery: Using specialized enhancers (short ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Scientists developed a promising gene replacement therapy for Dravet syndrome in mice. The therapy alleviated symptoms ...

Listed below are links to the GEN stories reference in this episode of Touching Base Gene Therapy Tested in Mice Offers New ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without side effects.

SEATTLE, Wash. -- Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is a rare and debilitating form of epilepsy that starts ...

New research from the University of Colorado Anschutz Medical Campus is the first to identify defects related to metabolism (how the body processes and uses energy) in lymphoblast cell lines (LCLs) ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form of epilepsy, scientists have developed a new gene replacement therapy ...