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Haematologica|September 14, 2014
Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemiaPekka Jaako, Shubhranshu Debnath, Karin Olsson, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 13, 2008
Physiological promoters reduce the genotoxic risk of integrating gene vectorsDaniela Zychlinski, Axel Schambach, Ute Modlich, et al.
Biomaterials|December 4, 2018
Sustained and regulated gene expression by Tet-inducible "all-in-one" retroviral vectors containing the HNRPA2B1-CBX3 UCOE®Katharina Cullmann, Kaj E C Blokland, Attila Sebe, et al.
Molecular Therapy. Nucleic Acids|February 4, 2022
Targeting transgenic proteins to alpha granules for platelet-directed gene therapyVanessa M A Woods, Lisette J Latorre-Rey, Franziska Schenk, et al.
Experimental Hematology|May 18, 2020
Signaling properties of murine MPL and MPL mutants after stimulation with thrombopoietin and romiplostimMarcel G E Rommel, Keven Hoerster, Christian Milde, et al.
Human Gene Therapy Methods|February 7, 2013
Evaluating a ligation-mediated PCR and pyrosequencing method for the detection of clonal contribution in polyclonal retrovirally transduced samplesMartijn H Brugman, Julia D Suerth, Michael Rothe, et al.
Blood|February 4, 2011
Lentiviral gene transfer regenerates hematopoietic stem cells in a mouse model for Mpl-deficient aplastic anemiaDirk Heckl, Daniel C Wicke, Martijn H Brugman, et al.
Hepatology (Baltimore, Md.)|December 22, 2012
Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodentsIna Rittelmeyer, Michael Rothe, Martijn H Brugman, et al.
Plos One|July 7, 2015
Inhibition of Thrombopoietin/Mpl Signaling in Adult Hematopoiesis Identifies New Candidates for Hematopoietic Stem Cell MaintenanceSaskia Kohlscheen, Sabine Wintterle, Adrian Schwarzer, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 27, 2009
Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control regionParitha I Arumugam, Tomoyasu Higashimoto, Fabrizia Urbinati, et al.
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