| Highly efficient therapeutic gene editing of human hematopoietic stem cells Y Wu, J Zeng, BP Roscoe, P Liu, Q Yao, CR Lazzarotto, K Clement, ... Nature medicine 25 (5), 776-783, 2019 | 430 | 2019 |
| Selective inhibition of tumor growth by clonal NK cells expressing an ErbB2/HER2-specific chimeric antigen receptor K Schönfeld, C Sahm, C Zhang, S Naundorf, C Brendel, M Odendahl, ... Molecular therapy 23 (2), 330-338, 2015 | 332 | 2015 |
| Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease EB Esrick, LE Lehmann, A Biffi, M Achebe, C Brendel, MF Ciuculescu, ... New England Journal of Medicine 384 (3), 205-215, 2021 | 316 | 2021 |
| Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype C Brendel, S Guda, R Renella, DE Bauer, MC Canver, YJ Kim, ... The Journal of clinical investigation 126 (10), 3868-3878, 2016 | 165 | 2016 |
| Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells G Santilli, E Almarza, C Brendel, U Choi, C Beilin, MP Blundell, S Haria, ... Molecular Therapy 19 (1), 122-132, 2011 | 161 | 2011 |
| miRNA-embedded shRNAs for lineage-specific BCL11A knockdown and hemoglobin F induction S Guda, C Brendel, R Renella, P Du, DE Bauer, MC Canver, JK Grenier, ... Molecular Therapy 23 (9), 1465-1474, 2015 | 133 | 2015 |
| Mammary epithelial reconstitution with gene-modified stem cells assigns roles to Stat5 in luminal alveolar cell fate decisions, differentiation, involution, and mammary tumor … V Vafaizadeh, P Klemmt, C Brendel, K Weber, C Doebele, K Britt, M Grez, ... Stem cells 28 (5), 928-938, 2010 | 104 | 2010 |
| Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome AL Kennedy, KC Myers, J Bowman, CJ Gibson, ND Camarda, E Furutani, ... Nature communications 12 (1), 1334, 2021 | 103 | 2021 |
| A ubiquitous chromatin opening element prevents transgene silencing in pluripotent stem cells and their differentiated progeny N Pfaff, N Lachmann, M Ackermann, S Kohlscheen, C Brendel, T Maetzig, ... Stem Cells 31 (3), 488-499, 2013 | 89 | 2013 |
| A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and … U Müller-Kuller, M Ackermann, S Kolodziej, C Brendel, J Fritsch, ... Nucleic acids research 43 (3), 1577-1592, 2015 | 88 | 2015 |
| Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions M Weisser, UM Demel, S Stein, L Chen-Wichmann, F Touzot, G Santilli, ... Journal of Allergy and Clinical Immunology 138 (1), 219-228. e9, 2016 | 76 | 2016 |
| Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia S Xu, K Luk, Q Yao, AH Shen, J Zeng, Y Wu, HY Luo, C Brendel, L Pinello, ... Blood, The Journal of the American Society of Hematology 133 (21), 2255-2262, 2019 | 75 | 2019 |
| Survival of the fittest: positive selection of CD4+ T cells expressing a membrane-bound fusion inhibitor following HIV-1 infection J Kimpel, SE Braun, G Qiu, FE Wong, M Conolle, JE Schmitz, C Brendel, ... PloS one 5 (8), e12357, 2010 | 68 | 2010 |
| Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter C Brendel, U Müller-Kuller, S Schultze-Strasser, S Stein, ... Gene therapy 19 (10), 1018-1029, 2012 | 61 | 2012 |
| Activating c-KIT mutations confer oncogenic cooperativity and rescue RUNX1/ETO-induced DNA damage and apoptosis in human primary CD34+ hematopoietic progenitors C Wichmann, I Quagliano-Lo Coco, Ö Yildiz, L Chen-Wichmann, H Weber, ... Leukemia 29 (2), 279-289, 2015 | 55 | 2015 |
| Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing S Knight, F Zhang, U Mueller-Kuller, M Bokhoven, A Gupta, T Broughton, ... Journal of virology 86 (17), 9088-9095, 2012 | 52 | 2012 |
| Preclinical evaluation of a novel lentiviral vector driving lineage-specific BCL11A knockdown for sickle cell gene therapy C Brendel, O Negre, M Rothe, S Guda, G Parsons, C Harris, ... Molecular Therapy-Methods & Clinical Development 17, 589-600, 2020 | 49 | 2020 |
| Surface functionalization of orthopedic titanium implants with bone sialoprotein A Baranowski, A Klein, U Ritz, A Ackermann, J Anthonissen, ... PLoS One 11 (4), e0153978, 2016 | 47 | 2016 |
| Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease C Brendel, M Rothe, G Santilli, S Charrier, S Stein, H Kunkel, D Abriss, ... Human Gene Therapy Clinical Development 29 (2), 69-79, 2018 | 41 | 2018 |
| Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells D Klatt, E Cheng, D Hoffmann, G Santilli, AJ Thrasher, C Brendel, ... Human Gene Therapy 31 (3-4), 199-210, 2020 | 39 | 2020 |
