Biblio

Author Title [ Type(Desc)] Year
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Journal Article
Nasri M, Ritter M, Mir P, Dannenmann B, Kaufmann MM, Arreba-Tutusaus P, Xu Y, Borbaran-Bravo N, Klimiankou M, Lengerke C, et al. CRISPR/Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia. Mol Ther. 2024.
Merkert S, Wunderlich S, Bednarski C, Beier J, Haase A, Dreyer A-K, Schwanke K, Meyer J, Göhring G, Cathomen T, et al. Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cells. Stem Cell Reports. 2014;2(1):107-18.
Marone R, Landmann E, Devaux A, Lepore R, Seyres D, Zuin J, Burgold T, Engdahl C, Capoferri G, Dell'Aglio A, et al. Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy. J Exp Med. 2023;220(12).
Turchiano G, Andrieux G, Klermund J, Blattner G, Pennucci V, Gaz MEl, Monaco G, Poddar S, Mussolino C, Cornu TI, et al. Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq. Cell Stem Cell. 2021.
Rahman SH, Kuehle J, Reimann C, Mlambo T, Alzubi J, Maeder ML, Riedel H, Fisch P, Cantz T, Rudolph C, et al. Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model. PLoS Genet. 2015;11(5):e1005239.
Dreyer A-K, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, Siler U, Reichenbach J, Grez M, Moritz T, et al. TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells. Biomaterials. 2015;69:191-200.
Bednarski C, Tomczak K, Hövel BVom, Weber W-M, Cathomen T. Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model. PLoS One. 2016;11(8):e0161072.