SCIE.05.146
ADENOSINE DEAMINASE 2 DEFICIENCY: FROM THE UNDERLYING DISEASE MECHANISMS TO GENE THERAPY
SCIE.11.156
HUMAN HEMATOPOIETIC STEM/PROGENITOR CELL TRAFFICKING AND CLONAL TRACKING
SCIE.11.157
DECONVOLUTING THE DYNAMICS OF HEMATOPOIETIC RECONSTITUTION IN GENE THERAPY PATIENTS
SCIE.11.195
GENERATION, SELECTION AND CHARACTERISATION OF A NOVEL MOUSE MODEL FOR MUCOPOLYSACCHARIDOSIS TYPE IVA
SCIE.11.196
AN INNOVATIVE PLATFORM APPROACH FOR THE DEVELOPMENT OF EX-VIVO GENE THERAPIES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH SKELETAL INVOLVEMENT