In this study, we utilized an approach similar to that of GAN GT to evaluate the efficacy and safety of MFSD8 gene transfer in vitro in fibroblasts from a CLN7 patient as well as in KO mouse models to investigate whether this would show a potential benefit to pediatric patients suffering from CLN7 disease. This evidence concerns the gene MFSD8 and neuronal ceroid lipofuscinosis 7.