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...

Celý popis

Uloženo v:
Podrobná bibliografie
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2013
Témata:
Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3538318/
https://ncbi.nlm.nih.gov/pubmed/22371846
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.23
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky