Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease
Sophia-Martha kleine Holthaus, Saúl Herranz-Martín, Giulia Massaro, Mikel Aristorena, Justin Hoke, Michael P. Hughes, Ryea N. Maswood, Olha Semenyuk, Mark Basche, Amna Z. Shah, Izabela P. Klaska, Alexander J. Smith, Sara Mole, Ahad A. Rahim, 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.
Human Molecular Genetics · Aug 28, 2019Peer-reviewedNeuroscience