University of Virginia School of Medicine scientists have used a next-generation form of gene editing to fix the underlying cause of a severe form of epilepsy in lab mice.

Baby KJ was a pioneer before he was even a year old. Born in 2024 with a rare, life-threatening genetic mutation, Nicole and ...

CRISPR Cas9 gene therapy explained with DNA scissors, hereditary diseases treatment, and designer babies ethical dilemmas ...

A single infusion of a CRISPR-based gene-editing therapy was associated with reductions in LDL cholesterol and triglycerides ...

Add Yahoo as a preferred source to see more of our stories on Google. Gene editing fixed brain mutations in mice with AHC, offering hope for treating rare neurological diseases at the DNA level.

Cross-species research shows that RNA splicing patterns, not just gene activity, track maximum lifespan in mammals, revealing ...

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a ...

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...

In May 2025, the world celebrated the success of KJ, an infant who was treated with the world’s first personalized CRISPR gene editing therapy. KJ was born with severe carbamoyl phosphate synthetase 1 ...

Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol.It's very early-stage research, tried in only a few ...

Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin ob ...