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Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.
Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A, Klatt D, Bentler M, Pellin D, Christiansen L, Schambach A, Manis J, Trebeden-Negre H, Bonner M, Esrick E, Veres G, Armant M, Williams DA. Brendel C, et al. Mol Ther Methods Clin Dev. 2020 Mar 17;17:589-600. doi: 10.1016/j.omtm.2020.03.015. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32300607 Free PMC article.
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: brendel c. 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.
Unexpected help: mTOR meets lentiviral vectors.
Brendel C, Williams DA. Brendel C, et al. Blood. 2014 Aug 7;124(6):832-3. doi: 10.1182/blood-2014-06-582411. Blood. 2014. PMID: 25104861 Free PMC article.
A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells.
Müller-Kuller U, Ackermann M, Kolodziej S, Brendel C, Fritsch J, Lachmann N, Kunkel H, Lausen J, Schambach A, Moritz T, Grez M. Müller-Kuller U, et al. Among authors: brendel c. Nucleic Acids Res. 2015 Feb 18;43(3):1577-92. doi: 10.1093/nar/gkv019. Epub 2015 Jan 20. Nucleic Acids Res. 2015. PMID: 25605798 Free PMC article.
miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction.
Guda S, Brendel C, Renella R, Du P, Bauer DE, Canver MC, Grenier JK, Grimson AW, Kamran SC, Thornton J, de Boer H, Root DE, Milsom MD, Orkin SH, Gregory RI, Williams DA. Guda S, et al. Among authors: brendel c. Mol Ther. 2015 Sep;23(9):1465-74. doi: 10.1038/mt.2015.113. Epub 2015 Jun 17. Mol Ther. 2015. PMID: 26080908 Free PMC article.
156 results