Ontology highlight
ABSTRACT:
SUBMITTER: Azario I
PROVIDER: S-EPMC5573317 | biostudies-literature | 2017 Aug
REPOSITORIES: biostudies-literature
Azario Isabella I Pievani Alice A Del Priore Federica F Antolini Laura L Santi Ludovica L Corsi Alessandro A Cardinale Lucia L Sawamoto Kazuki K Kubaski Francyne F Gentner Bernhard B Bernardo Maria Ester ME Valsecchi Maria Grazia MG Riminucci Mara M Tomatsu Shunji S Aiuti Alessandro A Biondi Andrea A Serafini Marta M
Scientific reports 20170825 1
Umbilical cord blood (UCB) is a promising source of stem cells to use in early haematopoietic stem cell transplantation (HSCT) approaches for several genetic diseases that can be diagnosed at birth. Mucopolysaccharidosis type I (MPS-I) is a progressive multi-system disorder caused by deficiency of lysosomal enzyme α-L-iduronidase, and patients treated with allogeneic HSCT at the onset have improved outcome, suggesting to administer such therapy as early as possible. Given that the best character ...[more]