Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-VivoFANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, Maria Grazia Roncarolo,Brian C. Beard, Ken Law,Eileen Nicoletti,Paula Rio,Juan A. Bueren,Jonathan D. Schwartz,Sandeep Soni

Blood（2019）

Cited 4|Views35
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Epigenetic Remodeling
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