Peripheral blood stem and progenitor cell collection in pediatric candidates for
apheresis
congenital
harvest
hematopoietic stem and progenitor cells
lenograstim
mobilization
plerixafor
rare disease
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
10 Sep 2021
10 Sep 2021
Historique:
received:
08
04
2021
accepted:
26
05
2021
entrez:
6
9
2021
pubmed:
7
9
2021
medline:
7
9
2021
Statut:
epublish
Résumé
Hematopoietic stem and progenitor cell (HSPC)-based gene therapy (GT) requires the collection of a large number of cells. While bone marrow (BM) is the most common source of HSPCs in pediatric donors, the collection of autologous peripheral blood stem cells (PBSCs) is an attractive alternative for GT. We present safety and efficacy data of a 10-year cohort of 45 pediatric patients who underwent PBSC collection for backup and/or purification of CD34
Identifiants
pubmed: 34485596
doi: 10.1016/j.omtm.2021.05.013
pii: S2329-0501(21)00097-8
pmc: PMC8390560
doi:
Types de publication
Journal Article
Langues
eng
Pagination
76-83Informations de copyright
© 2021.
Déclaration de conflit d'intérêts
SR-TIGET is a joint venture between Fondazione Telethon and OSR. Gene therapies for ADA-SCID, WAS, MLD, β-thalassemia, and MPSIH developed at SR-TIGET were licensed to Orchard Therapeutics (OTL) in 2018 and 2019. A.A. is the principal investigator (PI) of the above clinical trials. M.E.B. is the current PI of the MPSIH clinical trial.
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