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Gene editing of hematopoietic stem cells restores T-cell response in familial hemophagocytic lymphohistiocytosis.
Dettmer-Monaco V, Weißert K, Ammann S, Monaco G, Lei L, Gräßel L, Rhiel M, Rositzka J, Kaufmann MM, Geiger K, Andrieux G, Lao J, Thoulass G, Schell C, Boerries M, Illert AL, Cornu TI, Ehl S, Aichele P, Cathomen T. Dettmer-Monaco V, et al. Among authors: cathomen t. J Allergy Clin Immunol. 2024 Jan;153(1):243-255.e14. doi: 10.1016/j.jaci.2023.08.003. Epub 2023 Aug 16. J Allergy Clin Immunol. 2024. PMID: 37595758 Free article.
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies.
Venkatesan V, Christopher AC, Rhiel M, Azhagiri MKK, Babu P, Walavalkar K, Saravanan B, Andrieux G, Rangaraj S, Srinivasan S, Karuppusamy KV, Jacob A, Bagchi A, Pai AA, Nakamura Y, Kurita R, Balasubramanian P, Pai R, Marepally SK, Mohankumar KM, Velayudhan SR, Boerries M, Notani D, Cathomen T, Srivastava A, Thangavel S. Venkatesan V, et al. Among authors: cathomen t. Mol Ther Nucleic Acids. 2023 Apr 26;32:671-688. doi: 10.1016/j.omtn.2023.04.024. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37215154 Free PMC article.
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells.
Klermund J, Rhiel M, Kocher T, Chmielewski KO, Bischof J, Andrieux G, El Gaz M, Hainzl S, Boerries M, Cornu TI, Koller U, Cathomen T. Klermund J, et al. Among authors: cathomen t. Mol Ther. 2024 May 1;32(5):1298-1310. doi: 10.1016/j.ymthe.2024.03.006. Epub 2024 Mar 7. Mol Ther. 2024. PMID: 38459694 Free article.
High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis.
Papaioannou NY, Patsali P, Naiisseh B, Papasavva PL, Koniali L, Kurita R, Nakamura Y, Christou S, Sitarou M, Mussolino C, Cathomen T, Kleanthous M, Lederer CW. Papaioannou NY, et al. Among authors: cathomen t. Front Genome Ed. 2023 Mar 8;5:1141618. doi: 10.3389/fgeed.2023.1141618. eCollection 2023. Front Genome Ed. 2023. PMID: 36969374 Free PMC article.
Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectors.
Bexte T, Botezatu L, Miskey C, Gierschek F, Moter A, Wendel P, Reindl LM, Campe J, Villena-Ossa JF, Gebel V, Stein K, Cathomen T, Cremer A, Wels WS, Hudecek M, Ivics Z, Ullrich E. Bexte T, et al. Among authors: cathomen t. Mol Ther. 2024 May 14:S1525-0016(24)00320-4. doi: 10.1016/j.ymthe.2024.05.022. Online ahead of print. Mol Ther. 2024. PMID: 38751112
154 results