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Page 1
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: cornu ti. 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.
Correction: Patients and mice with deficiency in the SNARE protein SYNTAXIN-11 have a secondary B cell defect.
Kögl T, Chang HF, Staniek J, Chiang SCC, Thoulass G, Lao J, Weißert K, Dettmer-Monaco V, Geiger K, Manna PT, Beziat V, Momenilandi M, Tu SM, Keppler SJ, Pattu V, Wolf P, Kupferschmid L, Tholen S, Covill LE, Ebert K, Straub T, Groß M, Gather R, Engel H, Salzer U, Schell C, Maier S, Lehmberg K, Cornu TI, Pircher H, Shahrooei M, Parvaneh N, Elling R, Rizzi M, Bryceson YT, Ehl S, Aichele P, Ammann S. Kögl T, et al. Among authors: cornu ti. J Exp Med. 2024 Jul 1;221(7):e2022112205142024c. doi: 10.1084/jem.2022112205142024c. Epub 2024 May 21. J Exp Med. 2024. PMID: 38771261 Free PMC article. No abstract available.
Patients and mice with deficiency in the SNARE protein SYNTAXIN-11 have a secondary B cell defect.
Kögl T, Chang HF, Staniek J, Chiang SCC, Thoulass G, Lao J, Weißert K, Dettmer-Monaco V, Geiger K, Manna PT, Beziat V, Momenilandi M, Tu SM, Keppler SJ, Pattu V, Wolf P, Kupferschmid L, Tholen S, Covill LE, Ebert K, Straub T, Groß M, Gather R, Engel H, Salzer U, Schell C, Maier S, Lehmberg K, Cornu TI, Pircher H, Shahrooei M, Parvaneh N, Elling R, Rizzi M, Bryceson YT, Ehl S, Aichele P, Ammann S. Kögl T, et al. Among authors: cornu ti. J Exp Med. 2024 Jul 1;221(7):e20221122. doi: 10.1084/jem.20221122. Epub 2024 May 9. J Exp Med. 2024. PMID: 38722309 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: cornu ti. 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.
CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells.
Hammad R, Alzubi J, Rhiel M, Chmielewski KO, Mosti L, Rositzka J, Heugel M, Lawrenz J, Pennucci V, Gläser B, Fischer J, Schambach A, Moritz T, Lachmann N, Cornu TI, Mussolino C, Schäfer R, Cathomen T. Hammad R, et al. Among authors: cornu ti. Int J Mol Sci. 2024 Jan 12;25(2):985. doi: 10.3390/ijms25020985. Int J Mol Sci. 2024. PMID: 38256061 Free PMC article.
Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy.
Marone R, Landmann E, Devaux A, Lepore R, Seyres D, Zuin J, Burgold T, Engdahl C, Capoferri G, Dell'Aglio A, Larrue C, Simonetta F, Rositzka J, Rhiel M, Andrieux G, Gallagher DN, Schröder MS, Wiederkehr A, Sinopoli A, Do Sacramento V, Haydn A, Garcia-Prat L, Divsalar C, Camus A, Xu L, Bordoli L, Schwede T, Porteus M, Tamburini J, Corn JE, Cathomen T, Cornu TI, Urlinger S, Jeker LT. Marone R, et al. Among authors: cornu ti. J Exp Med. 2023 Dec 4;220(12):e20231235. doi: 10.1084/jem.20231235. Epub 2023 Sep 29. J Exp Med. 2023. PMID: 37773046 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: cornu ti. 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.
38 results