Hematopoietic Stem Cell Transplantation with Mesenchymal Stromal Cells in Children with Metachromatic Leukodystrophy.
GMFCS-MLD
aryl-sulfatase A
hematopoietic stem cell transplantation
mesenchymal stem cells
mesenchymal stromal cells
metachromatic leukodystrophy
Journal
Stem cells and development
ISSN: 1557-8534
Titre abrégé: Stem Cells Dev
Pays: United States
ID NLM: 101197107
Informations de publication
Date de publication:
04 2022
04 2022
Historique:
pubmed:
25
3
2022
medline:
26
4
2022
entrez:
24
3
2022
Statut:
ppublish
Résumé
Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder primarily affecting the white matter of the nervous system that results from a deficiency of the arylsulfatase A (ARSA). Mesenchymal stem cells (MSCs) are able to secrete ARSA and have shown beneficial effects in MLD patients. In this retrospective analysis, 10 pediatric MLD patients [mesenchymal stem cell group (MSCG)] underwent allogeneic hematopoietic stem cell transplantation (HSCT) and received two applications of 2 × 10
Identifiants
pubmed: 35323019
doi: 10.1089/scd.2021.0352
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM