Liver cancer is one of the world's deadliest cancers, and most cases are linked to chronic viral hepatitis. Yet scientists have lacked an animal model that faithfully recapitulates how the disease ...

Myofibrillar myopathy type 6 (MFM6) is a rare genetic muscle disorder that leads to severe muscle weakness and a drastically shortened life expectancy due to a disruption in muscle protein regulation.

Researchers at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Baylor College of Medicine recently made an important advance in neurodegenerative disease ...

Base editing approach reverses pathogenic SCN8A mutation in mice, with reduced seizure burden, improved survival and restored neuronal function pointing towards future gene therapies for severe ...

Medical researchers have used animals, particularly mice, to mimic disease and study biological phenomena. The origin of mice as a standard model system for scientific research can extend back to the ...

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his ...