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125 results
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Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
Patsali P, Turchiano G, Papasavva P, Romito M, Loucari CC, Stephanou C, Christou S, Sitarou M, Mussolino C, Cornu TI, Antoniou MN, Lederer CW, Cathomen T, Kleanthous M. Patsali P, et al. Among authors: cathomen t. Haematologica. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19. Haematologica. 2019. PMID: 31004018 Free PMC article. No abstract available.
TALEN mediated gene editing in a mouse model of Fanconi anemia.
Pino-Barrio MJ, Giménez Y, Villanueva M, Hildenbeutel M, Sánchez-Dominguez R, Rodríguez-Perales S, Pujol R, Surrallés J, Río P, Cathomen T, Mussolino C, Bueren JA, Navarro S. Pino-Barrio MJ, et al. Among authors: cathomen t. Sci Rep. 2020 Apr 24;10(1):6997. doi: 10.1038/s41598-020-63971-z. Sci Rep. 2020. PMID: 32332829 Free PMC article.
AAV vectors for gene correction.
Cathomen T. Cathomen T. Curr Opin Mol Ther. 2004 Aug;6(4):360-6. Curr Opin Mol Ther. 2004. PMID: 15468594 Review.
Custom zinc-finger nucleases for use in human cells.
Alwin S, Gere MB, Guhl E, Effertz K, Barbas CF 3rd, Segal DJ, Weitzman MD, Cathomen T. Alwin S, et al. Among authors: cathomen t. Mol Ther. 2005 Oct;12(4):610-7. doi: 10.1016/j.ymthe.2005.06.094. Mol Ther. 2005. PMID: 16039907
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