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TITLE: Evaluation of a CSF biomarker for gene therapy that discriminates neuronopathic and non-neuronopathic forms of MPS II
AUTHORS: Boulos, N.; Wu, Z.; Cho, Y.; Mulatya, C.; Fuller, M.; Ausseil, J.; Pavone, L. M.; Giugliani, R.; Fiscella, M.; Nevoret, M.;
PUBLISHED: 2021, SOURCE: HUMAN GENE THERAPY, VOLUME: 32, ISSUE: 19-20
INDEXED IN: WOS