Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease. - Johnson TB; Brudvig JJ; Likhite S; Pratt MA; White KA; Cain JT; Booth CD; Timm DJ; Davis SS; Meyerink B; Pineda R; Dennys-Rivers C; et al. (Frontiers in genetics, 2023)