| http://purl.uniprot.org/citations/28842642 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/28842642 | http://www.w3.org/2000/01/rdf-schema#comment | "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 characterized MPS-I murine model is an immunocompetent mouse, we here developed a transplantation system based on murine UCB. With the final aim of testing the therapeutic efficacy of UCB in MPS-I mice transplanted at birth, we first defined the features of murine UCB cells and demonstrated that they are capable of multi-lineage haematopoietic repopulation of myeloablated adult mice similarly to bone marrow cells. We then assessed the effectiveness of murine UCB cells transplantation in busulfan-conditioned newborn MPS-I mice. Twenty weeks after treatment, iduronidase activity was increased in visceral organs of MPS-I animals, glycosaminoglycans storage was reduced, and skeletal phenotype was ameliorated. This study explores a potential therapy for MPS-I at a very early stage in life and represents a novel model to test UCB-based transplantation approaches for various diseases."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.org/dc/terms/identifier | "doi:10.1038/s41598-017-09958-9"xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Santi L."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Aiuti A."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Sawamoto K."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Tomatsu S."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Antolini L."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Corsi A."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Riminucci M."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Biondi A."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Kubaski F."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Serafini M."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Valsecchi M.G."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Bernardo M.E."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Gentner B."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Azario I."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Pievani A."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Cardinale L."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/author | "Del Priore F."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/date | "2017"xsd:gYear |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/name | "Sci Rep"xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/pages | "9473"xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/title | "Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I."xsd:string |
| http://purl.uniprot.org/citations/28842642 | http://purl.uniprot.org/core/volume | "7"xsd:string |