Retrovirus-mediated transfer of the cDNA for human glucocerebrosidase into peripheral blood repopulating cells of patients with Gaucher's disease

The purpose of this study is to investigate whether ex vivo retrovirus-mediated transfer of the cDNA for human glucocerebrosidase (GC) into peripheral blood repopulating cells of patients with Type 1 Gaucher's disease followed by infusion of the transduced cells into the patient is safe and abl...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Human gene therapy 1997-11, Vol.8 (17), p.2143-2160
Hauptverfasser: Schuening, F, Longo, W L, Atkinson, M E, Zaboikin, M, Kiem, H P, Sanders, J, Scott, C R, Storb, R, Miller, A D, Reynolds, T, Bensinger, W, Rowley, S, Gooley, T, Darovsky, B, Appelbaum, F
Format: Artikel
Sprache:eng
Schlagworte:
Online-Zugang:Volltext
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
Beschreibung
Zusammenfassung:The purpose of this study is to investigate whether ex vivo retrovirus-mediated transfer of the cDNA for human glucocerebrosidase (GC) into peripheral blood repopulating cells of patients with Type 1 Gaucher's disease followed by infusion of the transduced cells into the patient is safe and able to improve or cure the disease. Peripheral blood repopulating cells (PBRC) will be mobilized by treatment of the patient with recombinant human granulocyte colony stimulating factor (rhG-CSF), collected by repeated leukapheresis and selected for CD34 positive cells by avidin-biotin-immunoadsorption. CD34 positive cells will then be transduced ex vivo over a 5-day period in a long-term marrow culture system containing medium with retrovirus supernatant. After transduction, cells will be infused into the patient without myeloablative treatment.
ISSN:1043-0342
DOI:10.1089/hum.1997.8.17-2143