To accomplish this, we used as a model the adeno-associated virus (AAV) vector AAVrh.10CLN2 that is currently being tested as gene therapy for late infantile neuronal ceroid lipofuscinosis (LINCL, CLN2 disease, Batten disease), a uniformly fatal inherited pediatric lysosomal storage disorder (ClinicalTrials.gov identifier NCT01161576)20, 21, 22. Here, TPP1 is linked to neuronal ceroid lipofuscinosis.