An Irish baby has become one of the first patients in the world to receive an experimental gene therapy for the rare and fatal disorder Canavan disease (see BioNews 1315).
Canavan disease is a genetic condition caused by the absence of an enzyme needed for normal brain development. Affected babies typically appear healthy at birth but go on to experience severe neurological decline as the disease progresses.
The therapy aims to deliver a functional copy of the faulty gene directly into the brain. However, the researchers emphasise that such interventions remain experimental and are being carefully evaluated for safety and effectiveness.
See the Irish Times for more information.