Lentivirus-based Gene Therapy of Hematopoietic Stem Cells in Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome (WAS) is an inherited immunodeficiency caused by mutations in the gene encoding WASP, a protein regulating the cytoskeleton. Hematopoietic stem/progenitor cell (HSPC) transplants can be curative but, when matched donors are unavailable, infusion of autologous HSPCs modified...

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發表在:Science
Main Authors: Aiuti, Alessandro, Biasco, Luca, Scaramuzza, Samantha, Ferrua, Francesca, Cicalese, Maria Pia, Baricordi, Cristina, Dionisio, Francesca, Calabria, Andrea, Giannelli, Stefania, Castiello, Maria Carmina, Bosticardo, Marita, Evangelio, Costanza, Assanelli, Andrea, Casiraghi, Miriam, Di Nunzio, Sara, Callegaro, Luciano, Benati, Claudia, Rizzardi, Paolo, Pellin, Danilo, Di Serio, Clelia, Schmidt, Manfred, Von Kalle, Christof, Gardner, Jason, Mehta, Nalini, Neduva, Victor, Dow, David J., Galy, Anne, Miniero, Roberto, Finocchi, Andrea, Metin, Ayse, Banerjee, Pinaki, Orange, Jordan, Galimberti, Stefania, Valsecchi, Maria Grazia, Biffi, Alessandra, Montini, Eugenio, Villa, Anna, Ciceri, Fabio, Roncarolo, Maria Grazia, Naldini, Luigi
格式: Artigo
語言:Inglês
出版: 2013
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC4375961/
https://ncbi.nlm.nih.gov/pubmed/23845947
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/science.1233151
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