Base editing corrects mutation and slows motor neuron disease in mouse model

In a study published in Molecular Therapy Advances a team of researchers led by Professor Haruhisa Inoue (Center for iPS Cell Research and Application, Kyoto University) and Professor Yuishin Izumi ...
GEN

PAMless Base Editors Correct X-CGD Mutation Efficiently and Precisely

X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder characterized by high susceptibility to infections that results from genetic mutations in the cytochrome b-245 beta chain ...
Hosted on MSN

Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his life. But the scientific advances that made the groundbreaking treatment ...
GEN

CRISPR Base Editing Repairs Hard-to-Treat Cystic Fibrosis Mutation in Cell Models

Affecting an estimated 100,000 people globally, cystic fibrosis (CF) cases stem from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In the past several decades, ...
The Scientist

Base Editing Corrects Mutations for Cardiac Disease in Mice

Point mutations in the lamin A (LMNA) gene cause several human diseases, ranging from congenital muscular dystrophy to premature aging, collectively known as laminopathies, which affect skeletal ...
News Medical

Scientists use base editing to repair mitochondrial DNA mutations

In a step toward treating mitochondrial diseases, researchers in the Netherlands have successfully edited harmful mutations in mitochondrial DNA using a genetic tool known as a base editor. The ...
Medical Xpress

Altering the behavior of living mice by editing genes in the brain

Researchers at Fudan University and the Chinese Academy of Sciences, Shanghai, China, have conducted whole-brain genome editing targeted to correct a single-base mutation associated with autism ...