Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
Dravet syndrome affects around 1 in 15,700 children, and most cases are caused by mutations in the SCN1A gene. This gene ...
GEN - Genetic Engineering and Biotechnology News12d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
EurekAlert!13d
Breakthrough Dravet syndrome gene therapy in mice brings new hope to families
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Technology Networks12d
Gene Therapy Offers Hope for Dravet Syndrome Treatment
Scientists developed a promising gene replacement therapy for Dravet syndrome in mice. The therapy alleviated symptoms ...
KXLY 4 News11d
Groundbreaking gene therapy research in Washington could lead to treat children living with severe epilepsy
Dravet Syndrome impacts 1 in about every 15,700 infants in the U.S. Most cases are caused by a mutation of the SCN1A gene, which is important for proper brain cell function. Seizures people with ...
pharmaphorum4d
Biogen bags rights to epilepsy drug from Stoke in $550m deal
Zorevunersen is an investigational antisense oligonucleotide (ASO) that targets the SCN1A gene, which Biogen says is the underlying cause of most cases of Dravet syndrome, and is on the brink of ...
pharmaphorum2y
CAMP4 raises $100m to take lead RNA drugs into clinic
In around 80% of cases, Dravet syndrome is caused by defects in a gene coding for a sodium channel – SCN1A – which is required for the proper functioning of neurons in the brain. CAMP4's drug ...