Efficacy and Safety in Mice and Non-Hman Primates of CSF Delivered AVXS-201 for the Treatment of Rett Syndrome

Foust, K; Thomsen, G; Fugere, M; Braun, L; Nguyen, K; Solano, S; Lang, E; Do, J; Chang, TK; Hsieh, C; Powers, S; Miranda, C; Dennys-Rivers, C; Huffenberger, A; Rinaldi, F; Wein, N; Morales, P; Kaspar, A; Meyer, K; Kaspar, BK

MOLECULAR THERAPY, 2019; 27 (4): 188

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