Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 Cells for Correction of Fabry Disease.
Molecular Therapy - Methods & Clinical Development（2017）
关键词
lysosomal storage disease,α-galactosidase A,hematopoietic stem/progenitor cell,xenotransplantation,GMP production
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