Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells

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<i>LMO2</i> -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

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A. R. W. Schröder, Paul Shinn, Huaming Chen et al. · Cell · 2002 · 1.8K citations · Full text

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Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

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