The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Researchers have engineered a new class of adenine base editors that reduce unwanted bystander mutations by two to three times compared to the widely used ABE8e, while preserving the editing power ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

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 ...

A landmark study in China has shown that a base-editing therapy developed by CorrectSequence Therapeutics achieved stellar results in patients with the severe blood disorder beta-thalassaemia. The ex ...