gene therapy

South Korean researchers build gene switch controlled by electromagnetic fields

Researchers at Dongguk University in Seoul have created a gene switch that can be turned on and off inside living animals using electromagnetic fields, a development that could reshape how gene therapies are delivered. The study, published in Cell on…

Personalized gene therapy helps teen with epilepsy walk independently

Researchers at UC San Diego and Rady Children's Institute for Genomic Medicine have demonstrated that personalized gene therapy tailored to individual genetic mutations can dramatically reduce seizures and unlock developmental milestones in children with a severe form of epilepsy. The…

New gene therapy platform uses brain’s own fluid system to bypass blood-brain barrier

Researchers at the University of Rochester Medical Center have developed a gene therapy platform that exploits the brain's natural fluid transport network — the glymphatic system — to distribute engineered viral vectors broadly throughout brain tissue, offering a potential path…

CRISPR therapy shows promise in children as young as 5 with sickle cell disease

HCA Healthcare announced on Monday that research published in The New England Journal of Medicine demonstrates promising results from a CRISPR gene-editing therapy tested in children ages 5 to 11 with severe sickle cell disease and transfusion-dependent beta thalassemia, marking…

Single gene therapy shot extended mouse lifespan by 20%, study finds

A single injection of gene therapy extended the lifespan of elderly mice by more than 20%, according to a study published in Molecular Therapy by researchers at the Universitat Autònoma de Barcelona. The findings mark the first demonstration that a…

China’s RNA editing therapy treats muscular dystrophy in global first

A research team from Changping Laboratory and Peking University has published results in the journal Cell showing that a China-developed RNA editing platform successfully treated pediatric patients with Duchenne muscular dystrophy, marking the first clinical application of RNA editing for…

First person treated with cell-rejuvenating gene therapy for glaucoma

Life Biosciences on Monday announced that the first participant has been dosed in its Phase 1 clinical trial of ER-100, a gene therapy designed to reverse cellular aging in patients with vision loss — marking the first time partial epigenetic…