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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: bak ro. 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.
Efficient sleeping beauty DNA transposition from DNA minicircles.
Sharma N, Cai Y, Bak RO, Jakobsen MR, Schrøder LD, Mikkelsen JG. Sharma N, et al. Among authors: bak ro. Mol Ther Nucleic Acids. 2013 Feb 26;2(2):e74. doi: 10.1038/mtna.2013.1. Mol Ther Nucleic Acids. 2013. PMID: 23443502 Free PMC article.
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.
Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J, Kaushik M, Hay SR, Martin RM, Steinfeld I, Bak RO, Dever DP, Hendel A, Bruhn L, Porteus MH. Cromer MK, et al. Among authors: bak ro. Mol Ther. 2018 Oct 3;26(10):2431-2442. doi: 10.1016/j.ymthe.2018.06.002. Epub 2018 Jul 11. Mol Ther. 2018. PMID: 30005866 Free PMC article.
The p53 challenge of hematopoietic stem cell gene editing.
Dorset SR, Bak RO. Dorset SR, et al. Among authors: bak ro. Mol Ther Methods Clin Dev. 2023 Jun 12;30:83-89. doi: 10.1016/j.omtm.2023.06.003. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37435043 Free PMC article. Review.
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.
Hendel A, Bak RO, Clark JT, Kennedy AB, Ryan DE, Roy S, Steinfeld I, Lunstad BD, Kaiser RJ, Wilkens AB, Bacchetta R, Tsalenko A, Dellinger D, Bruhn L, Porteus MH. Hendel A, et al. Among authors: bak ro. Nat Biotechnol. 2015 Sep;33(9):985-989. doi: 10.1038/nbt.3290. Epub 2015 Jun 29. Nat Biotechnol. 2015. PMID: 26121415 Free PMC article.
72 results