Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome) - Commentary

Hunter syndrome is an X-linked metabolic storage disorder arising from defi ciency of iduronate sulfatase enzyme activity. Despite the successful use o f hematopoietic cell transplantation for a variety of lysosomal and peroxis omal storage diseases, limited benefit occurs following transplantation in either the severe or mild forms of Hunter syndrome. A brief ethical comment ary is provided on the ease of a boy with mucopolysaccharidosis IIB tie the mild form) who received an unrelated umbilical cord blood transplant to im prove his future quality of life.