New research demonstrates the long term safety and benefit of a virus-based gene therapy that has been applied for the first time in a clinical setting. This novel therapy was used to treat young patients with Canavan disease, a devastating inherited neurological condition that typically takes a child’s life by age ten. Results of the study have just been published in the journal Science Translational Medicine, in an article that describes this first clinical application of a viral-based gene therapy for a neurodegenerative/neurological disorder.
View the original article here: Novel virus-based gene therapy holds promise for Canavan disease
