Long-term therapeutic efficacy of intravenous AAV-mediated hamartin replacement in mouse model of tuberous sclerosis type 1. (September 2019) - Cheah, Pike See; Prabhakar, Shilpa; Zhang, Xuan; Zinter, Max; Gianatasio, Maria; Bronson, Roderick; Kwiatkowski, David; Stemmer-Rachamimov, Anat; Maguire, Casey; Sena-Esteves, Miguel; Tannous, Bakhos; Breakefield, Xandra