Gene Therapy for Metachromatic Leukodystrophy (MLD)

Exclusion Criteria

• •HIV RNA and/or HCV RNA and/or HBV DNA positive patients;
• •Patients affected by neoplastic diseases;
• •Patients with cytogenetic alterations typical of MDS/AML;
• •Patients with end-organ functions or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study;
• •Patients enrolled in other trials/other therapeutic approaches that might become available;
• •Patient who underwent allogeneic hematopoietic stem cell transplantation in the previous six months;
• •Patient who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.