NeuroscienceHuman Molecular GeneticsPeer-reviewed
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease
Sophia-Martha kleine Holthaus, Saúl Herranz-Martín, Giulia Massaro…·Robin R. Ali
This study shows that neonatal brain-directed gene therapy using AAV9-CLN6 significantly improves survival, motor function, and reduces neuropathology in a mouse model of CLN6 Batten disease.
31 citations