Long-term therapeutic efficacy of intravenous AAV-mediated hamartin replacement in mouse model of tuberous sclerosis type 1

by Prabhakar, Shilpa, Cheah, Pike See, Zhang, Xuan, Zinter, Max, Gianatasio, Maria, Hudry, Eloise, Bronson, Roderick T, Kwiatkowski, David J, Rachamimov, Anat Stemmer, Maguire, Casey A, Sena-Esteves, Miguel, Tannous, Bakhos A, Breakefield, Xandra O
Published 2019

Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2

by Prabhakar, Shilpa, Beauchamp, Roberta L., Cheah, Pike See, Yoshinaga, Akiko, Haidar, Edwina Abou, Lule, Sevda, Mani, Gayathri, Maalouf, Katia, Stemmer-Rachamimov, Anat, Jung, David H., Welling, D. Bradley, Giovannini, Marco, Plotkin, Scott R., Maguire, Casey A., Ramesh, Vijaya, Breakefield, Xandra O.
Published 2022