Prime Editing Repairs Five Genetic Mutations to Treat Rare Brain Disorder in Mice

Gene editing shown to correct rare brain mutations in mice

Scientists have used a single injection to correct gene mutations caused by an ultra-rare disease, improving symptoms and survival rates in mice. Published in Cell, the gene editing study targeted the 2 most common mutations that cause alternating hemiplegia in childhood (AHC). AHC is a rare neurological disorder affecting 1 in a million people. Symptoms, which usually begin before the age of 18 months, include weakness and paralysis in one or b…

Gene editing in the brain offers hope for ultra-rare neurological disease

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

Genome editing has corrected rare brain mutations in mice; could also help fight neurological diseases

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

Prime editing repairs five genetic mutations to treat rare brain disorder in mice

By the time they are a few months old, infants born with alternating hemiplegia of childhood (AHC) begin experiencing terrifying episodes of paralysis and seizures, and will soon show developmental delays and intellectual disability. There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.

Gene Editing Reverses Symptoms of Rare Brain Disorder in Mice

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

Prime Editing of Common Variants Reverses Pediatric Brain Disorder in Mice

A collaboration between gene editing pioneer David Liu, PhD, professor at the Broad Institute and Harvard University, and Cathleen Lutz, PhD, Vice President of Rare Disease Translational Center at the Jackson Laboratory (JAX), has demonstrated that in vivo prime editing can correct disease-causing mutations in mice modeling a rare pediatric brain disorder called Alternating Hemiplegia of Childhood (AHC). This severe neurodevelopmental disorder s…