Parameters affecting successful stem cell collections for genetic therapies in sickle cell disease

Emerging cellular therapies require the collection of peripheral blood hematopoietic stem cells (HSC) by apheresis for in vitro manipulation to accomplish gene addition or gene editing. These therapies require relatively large numbers of HSCs within a short time frame to generate an efficacious ther...

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Veröffentlicht in:	Transfusion and apheresis science 2021-02, Vol.60 (1), p.103059-103059, Article 103059
Hauptverfasser:	Justus, David G., Manis, John P.
Format:	Artikel
Sprache:	eng
Schlagworte:	Gene editing Gene therapy Health technology assessment Sickle cell disease Stem cells
Online-Zugang:	Volltext
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Beschreibung
Zusammenfassung:	Emerging cellular therapies require the collection of peripheral blood hematopoietic stem cells (HSC) by apheresis for in vitro manipulation to accomplish gene addition or gene editing. These therapies require relatively large numbers of HSCs within a short time frame to generate an efficacious therapeutic product. This review focuses on the principal factors that affect collection outcomes, especially relevant to gene therapy for sickle cell disease.
ISSN:	1473-0502 1878-1683
DOI:	10.1016/j.transci.2021.103059