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Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome.
DeKelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, Sancak Y, Cui X, Steine EJ, Miller JC, Tam P, Bartsevich VV, Meng X, Rupniewski I, Gopalan SM, Sun HC, Pitz KJ, Rock JM, Zhang L, Davis GD, Rebar EJ, Cheeseman IM, Yamamoto KR, Sabatini DM, Jaenisch R, Gregory PD, Urnov FD. DeKelver RC, et al. Genome Res. 2010 Aug;20(8):1133-42. doi: 10.1101/gr.106773.110. Epub 2010 May 27. Genome Res. 2010. PMID: 20508142 Free PMC article.
Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology.
Orlando SJ, Santiago Y, DeKelver RC, Freyvert Y, Boydston EA, Moehle EA, Choi VM, Gopalan SM, Lou JF, Li J, Miller JC, Holmes MC, Gregory PD, Urnov FD, Cost GJ. Orlando SJ, et al. Among authors: dekelver rc. Nucleic Acids Res. 2010 Aug;38(15):e152. doi: 10.1093/nar/gkq512. Epub 2010 Jun 8. Nucleic Acids Res. 2010. PMID: 20530528 Free PMC article.
Chromosomal translocations induced at specified loci in human stem cells.
Brunet E, Simsek D, Tomishima M, DeKelver R, Choi VM, Gregory P, Urnov F, Weinstock DM, Jasin M. Brunet E, et al. Proc Natl Acad Sci U S A. 2009 Jun 30;106(26):10620-5. doi: 10.1073/pnas.0902076106. Epub 2009 Jun 23. Proc Natl Acad Sci U S A. 2009. PMID: 19549848 Free PMC article.
Targeted transgene integration in plant cells using designed zinc finger nucleases.
Cai CQ, Doyon Y, Ainley WM, Miller JC, Dekelver RC, Moehle EA, Rock JM, Lee YL, Garrison R, Schulenberg L, Blue R, Worden A, Baker L, Faraji F, Zhang L, Holmes MC, Rebar EJ, Collingwood TN, Rubin-Wilson B, Gregory PD, Urnov FD, Petolino JF. Cai CQ, et al. Among authors: dekelver rc. Plant Mol Biol. 2009 Apr;69(6):699-709. doi: 10.1007/s11103-008-9449-7. Epub 2008 Dec 27. Plant Mol Biol. 2009. PMID: 19112554
Precise genome modification in the crop species Zea mays using zinc-finger nucleases.
Shukla VK, Doyon Y, Miller JC, DeKelver RC, Moehle EA, Worden SE, Mitchell JC, Arnold NL, Gopalan S, Meng X, Choi VM, Rock JM, Wu YY, Katibah GE, Zhifang G, McCaskill D, Simpson MA, Blakeslee B, Greenwalt SA, Butler HJ, Hinkley SJ, Zhang L, Rebar EJ, Gregory PD, Urnov FD. Shukla VK, et al. Among authors: dekelver rc. Nature. 2009 May 21;459(7245):437-41. doi: 10.1038/nature07992. Epub 2009 Apr 29. Nature. 2009. PMID: 19404259
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases.
Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC, Katibah GE, Amora R, Boydston EA, Zeitler B, Meng X, Miller JC, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Jaenisch R. Hockemeyer D, et al. Among authors: dekelver rc. Nat Biotechnol. 2009 Sep;27(9):851-7. doi: 10.1038/nbt.1562. Epub 2009 Aug 13. Nat Biotechnol. 2009. PMID: 19680244 Free PMC article.
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ, High KA. Sharma R, et al. Among authors: dekelver rc. Blood. 2015 Oct 8;126(15):1777-84. doi: 10.1182/blood-2014-12-615492. Epub 2015 Aug 21. Blood. 2015. PMID: 26297739 Free PMC article.
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets.
Conway A, Mendel M, Kim K, McGovern K, Boyko A, Zhang L, Miller JC, DeKelver RC, Paschon DE, Mui BL, Lin PJC, Tam YK, Barbosa C, Redelmeier T, Holmes MC, Lee G. Conway A, et al. Among authors: dekelver rc. Mol Ther. 2019 Apr 10;27(4):866-877. doi: 10.1016/j.ymthe.2019.03.003. Epub 2019 Mar 7. Mol Ther. 2019. PMID: 30902585 Free PMC article.
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
Laoharawee K, DeKelver RC, Podetz-Pedersen KM, Rohde M, Sproul S, Nguyen HO, Nguyen T, St Martin SJ, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T, McIvor RS. Laoharawee K, et al. Among authors: dekelver rc. Mol Ther. 2018 Apr 4;26(4):1127-1136. doi: 10.1016/j.ymthe.2018.03.002. Epub 2018 Mar 10. Mol Ther. 2018. PMID: 29580682 Free PMC article.
21 results