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BONE DEFECTS AND CROSS-CORRECTION IN MPSIH HSPC-GT

EX VIVO EXPANSION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS (HSPC) FOR GENE THERAPY

HUMAN HEMATOPOIETIC STEM/PROGENITOR CELL TRAFFICKING AND CLONAL TRACKING

DECONVOLUTING THE DYNAMICS OF HEMATOPOIETIC RECONSTITUTION IN GENE THERAPY PATIENTS

AN INNOVATIVE PLATFORM APPROACH FOR THE DEVELOPMENT OF EX-VIVO GENE THERAPIES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH SKELETAL INVOLVEMENT