Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions

M Aldenhoven, J Kurtzberg - Cytotherapy, 2015 - Elsevier
Initially used as an alternative hematopoietic stem cell source for patients without a human
leukocyte antigen–matched bone marrow or peripheral blood stem cell donor, unrelated
cord blood (UCB) is now the preferred donor source when hematopoietic stem cell
transplantation (HSCT) is used to treat patients with lysosomal storage disorders (LSD).
Without transplantation, these patients have serious progressive multi-system deterioration
and premature death. UCB transplantation favorably alters the natural history of these …