Brendel, Christian

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Wu, Y., Zeng, J., Roscoe, B. P., Liu, P., Yao, Q., Lazzarotto, C. R., Clement, K., Cole, M. A., Luk, K., Baricordi, C., et al. (2019) · Nature Medicine

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 515

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

Esrick, E. B., Lehmann, L. E., Biffi, A., Achebe, M., Brendel, C., Ciuculescu, M. F., Daley, H., MacKinnon, B., Morris, E., Federico, A., et al. (2021) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 449

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.

Brendel, C., Guda, S., Renella, R., Bauer, D. E., Canver, M. C., Kim, Y.-J., Heeney, M. M., Klatt, D., Fogel, J., Milsom, M. D., et al. (2016) · Journal of Clinical Investigation

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 167

Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions

Weisser, M., Demel, U. M., Stein, S., Chen-Wichmann, L., Touzot, F., Santilli, G., Sujer, S., Brendel, C., Siler, U., Cavazzana, M., et al. (2016) · Journal of Allergy and Clinical Immunology

Immunodeficiency and Autoimmune DisordersHematopoietic Stem Cell Transplantation

Cited by 77

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., et al. (2020) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 72