Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

Gene therapy with ex vivo-transduced hematopoietic stem/progenitor cells may represent a valid therapeutic option for monogenic immunohematological disorders such as Wiskott-Aldrich syndrome (WAS), a primary immunodeficiency associated with thrombocytopenia. We evaluated the preclinical safety and e...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	Molecular therapy 2013-01, Vol.21 (1), p.175-184
Hauptverfasser:	Scaramuzza, Samantha, Biasco, Luca, Ripamonti, Anna, Castiello, Maria C, Loperfido, Mariana, Draghici, Elena, Hernandez, Raisa J, Benedicenti, Fabrizio, Radrizzani, Marina, Salomoni, Monica, Ranzani, Marco, Bartholomae, Cynthia C, Vicenzi, Elisa, Finocchi, Andrea, Bredius, Robbert, Bosticardo, Marita, Schmidt, Manfred, von Kalle, Christof, Montini, Eugenio, Biffi, Alessandra, Roncarolo, Maria G, Naldini, Luigi, Villa, Anna, Aiuti, Alessandro
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Antigens, CD34 - immunology Biodistribution Blood Bone marrow Bone Marrow Cells - cytology Bone Marrow Cells - immunology Bone Marrow Transplantation Clinical trials Disease Gene therapy Genetic Vectors Good Manufacturing Practice Lentivirus - genetics Manufacturing Medical research Mice Mice, Knockout Original Patients Pediatrics Signal transduction Toxicity Transduction, Genetic Vectors (Biology) Wiskott-Aldrich Syndrome - therapy
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!