Article
From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.
Human gene therapy. Clinical development - 1 Jun 2013
Stein Stefan, Scholz Simone, Schwäble Joachim, Sadat Mohammed A, Modlich Ute, Schultze-Strasser Stephan, Diaz Margarita, Chen-Wichmann Linping, Müller-Kuller Uta, Brendel Christian, Fronza Raffaele, Kaufmann Kerstin B, Naundorf Sonja, Pech Nancy K, Travers Jeffrey B, Matute Juan D, Presson Robert G, Sandusky George E, Kunkel Hana, Rudolf Eva, Dillmann Adelina, von Kalle Christof, Kühlcke Klaus, Baum Christopher, Schambach Axel, Dinauer Mary C, Schmidt Manfred, Grez Manuel
Abstract excerpt
Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by impaired antimicrobial activity in phagocytic cells. As a monogenic disease affecting the hematopoietic system, CGD is amenable to gene therapy. Indeed in a phase I/II clinical trial, we demonstrated a transient re...
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