Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease

Nadia L. Mitchell, Samantha J. Murray, Martin P. Wellby, Graham K. Barrell, Katharina N. Russell, Ashley R. Deane, John R. Wynyard, Madeleine J. Palmer, Anila Pulickan, Phillipa M. Prendergast, Widler Casy, Steven J. Gray, David N. Palmer