Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease.
Mitchell NL, Russell KN, Wellby MP, Wicky HE, Schoderboeck L, Barrell GK, Melzer TR, Gray SJ, Hughes SM, Palmer DN. Mol Ther. 2018 Oct 3; 26(10):2366-2378. Epub 2018 Jul 17.