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From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.
Stein S, Scholz S, Schwäble J, Sadat MA, Modlich U, Schultze-Strasser S, Diaz M, Chen-Wichmann L, Müller-Kuller U, Brendel C, Fronza R, Kaufmann KB, Naundorf S, Pech NK, Travers JB, Matute JD, Presson RG Jr, Sandusky GE, Kunkel H, Rudolf E, Dillmann A, von Kalle C, Kühlcke K, Baum C, Schambach A, Dinauer MC, Schmidt M, Grez M. Stein S, et al. Among authors: grez m. Hum Gene Ther Clin Dev. 2013 Jun;24(2):86-98. doi: 10.1089/humc.2013.019. Hum Gene Ther Clin Dev. 2013. PMID: 23845071 Free PMC article.
CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells.
Brendel C, Goebel B, Daniela A, Brugman M, Kneissl S, Schwäble J, Kaufmann KB, Müller-Kuller U, Kunkel H, Chen-Wichmann L, Abel T, Serve H, Bystrykh L, Buchholz CJ, Grez M. Brendel C, et al. Among authors: grez m. Mol Ther. 2015 Jan;23(1):63-70. doi: 10.1038/mt.2014.173. Epub 2014 Sep 5. Mol Ther. 2015. PMID: 25189742 Free PMC article.
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.
Weisser M, Demel UM, Stein S, Chen-Wichmann L, Touzot F, Santilli G, Sujer S, Brendel C, Siler U, Cavazzana M, Thrasher AJ, Reichenbach J, Essers MAG, Schwäble J, Grez M. Weisser M, et al. Among authors: grez m. J Allergy Clin Immunol. 2016 Jul;138(1):219-228.e9. doi: 10.1016/j.jaci.2015.11.028. Epub 2016 Feb 4. J Allergy Clin Immunol. 2016. PMID: 26853280
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Brendel C, Rothe M, Santilli G, Charrier S, Stein S, Kunkel H, Abriss D, Müller-Kuller U, Gaspar B, Modlich U, Galy A, Schambach A, Thrasher AJ, Grez M. Brendel C, et al. Among authors: grez m. Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79. doi: 10.1089/humc.2017.245. Epub 2018 Apr 17. Hum Gene Ther Clin Dev. 2018. PMID: 29664709
140 results