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163 results

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Page 1
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.
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.
Klapwijk JC, Del Rio Espinola A, Libertini S, Collin P, Fellows MD, Jobling S, Lynch AM, Martus H, Vickers C, Zeller A, Biasco L, Brugman MH, Bushmann FD, Cathomen T, Ertl HCJ, Gabriel R, Gao G, Jadlowsky JK, Kimber I, Lanz TA, Levine BL, Micklethwaite KP, Onodera M, Pizzurro DM, Reed S, Rothe M, Sabatino DE, Salk JJ, Schambach A, Themis M, Yuan J. Klapwijk JC, et al. Among authors: cathomen t. Hum Gene Ther. 2024 Aug;35(15-16):527-542. doi: 10.1089/hum.2024.033. Hum Gene Ther. 2024. PMID: 39049734 Review.
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.
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.
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.
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.
Esposito F, Dell'Aquila F, Rhiel M, Auricchio S, Chmielewski KO, Andrieux G, Ferla R, Horrach PS, Padmanabhan A, Di Cunto R, Notaro S, Santeularia ML, Boerries M, Dell'Anno M, Nusco E, Padula A, Nutarelli S, Cornu TI, Sorrentino NC, Piccolo P, Trapani I, Cathomen T, Auricchio A. Esposito F, et al. Among authors: cathomen t. Cell Rep Med. 2024 Jul 16;5(7):101619. doi: 10.1016/j.xcrm.2024.101619. Epub 2024 Jun 18. Cell Rep Med. 2024. PMID: 38897206 Free PMC article.
Modulation of TCR stimulation and pifithrin-α improve the genomic safety profile of CRISPR-engineered human T cells.
Ursch LT, Müschen JS, Ritter J, Klermund J, Bernard BE, Kolb S, Warmuth L, Andrieux G, Miller G, Jiménez-Muñoz M, Theis FJ, Boerries M, Busch DH, Cathomen T, Schumann K. Ursch LT, et al. Among authors: cathomen t. Cell Rep Med. 2024 Dec 17;5(12):101846. doi: 10.1016/j.xcrm.2024.101846. Epub 2024 Dec 4. Cell Rep Med. 2024. PMID: 39637860 Free article.
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Cavazza A, Hendel A, Bak RO, Rio P, Güell M, Lainšček D, Arechavala-Gomeza V, Peng L, Hapil FZ, Harvey J, Ortega FG, Gonzalez-Martinez C, Lederer CW, Mikkelsen K, Gasiunas G, Kalter N, Gonçalves MAFV, Petersen J, Garanto A, Montoliu L, Maresca M, Seemann SE, Gorodkin J, Mazini L, Sanchez R, Rodriguez-Madoz JR, Maldonado-Pérez N, Laura T, Schmueck-Henneresse M, Maccalli C, Grünewald J, Carmona G, Kachamakova-Trojanowska N, Miccio A, Martin F, Turchiano G, Cathomen T, Luo Y, Tsai SQ, Benabdellah K; COST Action CA21113. Cavazza A, et al. Among authors: cathomen t. Mol Ther Nucleic Acids. 2023 Oct 29;34:102066. doi: 10.1016/j.omtn.2023.102066. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38034032 Free PMC article. Review.
163 results