SCIE.05.99
DISSECTING THE MECHANISMS OF MYELOID-TO-NEURAL ENZYMATIC CROSS-CORRECTION IN THE CONTEXT OF HEMATOPOIETIC STEM CELL GENE THERAPY FOR METACHROMATIC LEUKODYSTROPHY
SCIE.16.88
IN VITRO VALIDATION OF LENTIVIRAL VECTORS ENCODING FOR CHIMERIC MURINE AND HUMAN GALC ENZYMES TO IMPROVE THE EFFICACY OF GENE THERAPY APPROACHES FOR GLOBOID CELL LEUKODYSTROPHY