Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
12 2019
12 2019
Historique:
received:
09
11
2018
accepted:
05
04
2019
revised:
15
03
2019
pubmed:
1
6
2019
medline:
1
9
2020
entrez:
1
6
2019
Statut:
ppublish
Résumé
Collection of an adequate amount of autologous haematopoietic stem progenitor cells (HSPC) is required for ex vivo manipulation and successful engraftment for certain inherited disorders. Fifty-seven paediatric patients (age 0.5-11.4 years) underwent a bone marrow harvest for the purpose of HSPC gene therapy (GT), including adenosine deaminase-severe combined immunodeficiency (ADA-SCID), Wiskott-Aldrich syndrome (WAS) and metachromatic leukodystrophy (MLD) patients. Total nucleated cells and the percentage and absolute counts of CD34+ cells were calculated at defined steps of the procedure (harvest, CD34+ cell purification, transduction with the gene transfer vector and infusion of the medicinal product). A minimum CD34+ cell dose for infusion was 2 × 10
Identifiants
pubmed: 31150018
doi: 10.1038/s41409-019-0573-6
pii: 10.1038/s41409-019-0573-6
pmc: PMC6897559
mid: EMS82557
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1995-2003Subventions
Organisme : Telethon
ID : TGTGSKS1
Pays : Italy
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