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Items: 19

1.

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.

Mol Ther. 2019 Apr 10;27(4):866-877. doi: 10.1016/j.ymthe.2019.03.003. Epub 2019 Mar 7.

PMID:
30902585
2.

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.

Ou L, DeKelver RC, Rohde M, Tom S, Radeke R, St Martin SJ, Santiago Y, Sproul S, Przybilla MJ, Koniar BL, Podetz-Pedersen KM, Laoharawee K, Cooksley RD, Meyer KE, Holmes MC, McIvor RS, Wechsler T, Whitley CB.

Mol Ther. 2019 Jan 2;27(1):178-187. doi: 10.1016/j.ymthe.2018.10.018. Epub 2018 Nov 1.

3.

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.

Mol Ther. 2018 Apr 4;26(4):1127-1136. doi: 10.1016/j.ymthe.2018.03.002. Epub 2018 Mar 10.

4.

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.

Blood. 2015 Oct 8;126(15):1777-84. doi: 10.1182/blood-2014-12-615492. Epub 2015 Aug 21.

5.

SRSF2 Is Essential for Hematopoiesis, and Its Myelodysplastic Syndrome-Related Mutations Dysregulate Alternative Pre-mRNA Splicing.

Komeno Y, Huang YJ, Qiu J, Lin L, Xu Y, Zhou Y, Chen L, Monterroza DD, Li H, DeKelver RC, Yan M, Fu XD, Zhang DE.

Mol Cell Biol. 2015 Sep 1;35(17):3071-82. doi: 10.1128/MCB.00202-15. Epub 2015 Jun 29.

6.

Cooperation between RUNX1-ETO9a and novel transcriptional partner KLF6 in upregulation of Alox5 in acute myeloid leukemia.

DeKelver RC, Lewin B, Lam K, Komeno Y, Yan M, Rundle C, Lo MC, Zhang DE.

PLoS Genet. 2013;9(10):e1003765. doi: 10.1371/journal.pgen.1003765. Epub 2013 Oct 10.

7.

JAK inhibitors suppress t(8;21) fusion protein-induced leukemia.

Lo MC, Peterson LF, Yan M, Cong X, Hickman JH, Dekelver RC, Niewerth D, Zhang DE.

Leukemia. 2013 Dec;27(12):2272-9. doi: 10.1038/leu.2013.197. Epub 2013 Jul 1.

8.

RUNX1-ETO induces a type I interferon response which negatively effects t(8;21)-induced increased self-renewal and leukemia development.

DeKelver RC, Lewin B, Weng S, Yan M, Biggs J, Zhang DE.

Leuk Lymphoma. 2014 Apr;55(4):884-91. doi: 10.3109/10428194.2013.815351. Epub 2013 Jul 25.

9.

Attenuation of AML1-ETO cellular dysregulation correlates with increased leukemogenic potential.

DeKelver RC, Yan M, Ahn EY, Shia WJ, Speck NA, Zhang DE.

Blood. 2013 May 2;121(18):3714-7. doi: 10.1182/blood-2012-11-465641. Epub 2013 Feb 20.

10.

SON protein regulates GATA-2 through transcriptional control of the microRNA 23a~27a~24-2 cluster.

Ahn EE, Higashi T, Yan M, Matsuura S, Hickey CJ, Lo MC, Shia WJ, DeKelver RC, Zhang DE.

J Biol Chem. 2013 Feb 22;288(8):5381-8. doi: 10.1074/jbc.M112.447227. Epub 2013 Jan 14.

11.

Transcriptional activation of Brassica napus β-ketoacyl-ACP synthase II with an engineered zinc finger protein transcription factor.

Gupta M, DeKelver RC, Palta A, Clifford C, Gopalan S, Miller JC, Novak S, Desloover D, Gachotte D, Connell J, Flook J, Patterson T, Robbins K, Rebar EJ, Gregory PD, Urnov FD, Petolino JF.

Plant Biotechnol J. 2012 Sep;10(7):783-91. doi: 10.1111/j.1467-7652.2012.00695.x. Epub 2012 Apr 23.

12.

SON controls cell-cycle progression by coordinated regulation of RNA splicing.

Ahn EY, DeKelver RC, Lo MC, Nguyen TA, Matsuura S, Boyapati A, Pandit S, Fu XD, Zhang DE.

Mol Cell. 2011 Apr 22;42(2):185-98. doi: 10.1016/j.molcel.2011.03.014.

13.

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.

Nucleic Acids Res. 2010 Aug;38(15):e152. doi: 10.1093/nar/gkq512. Epub 2010 Jun 8.

14.

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.

Genome Res. 2010 Aug;20(8):1133-42. doi: 10.1101/gr.106773.110. Epub 2010 May 27.

15.

Distinct factors control histone variant H3.3 localization at specific genomic regions.

Goldberg AD, Banaszynski LA, Noh KM, Lewis PW, Elsaesser SJ, Stadler S, Dewell S, Law M, Guo X, Li X, Wen D, Chapgier A, DeKelver RC, Miller JC, Lee YL, Boydston EA, Holmes MC, Gregory PD, Greally JM, Rafii S, Yang C, Scambler PJ, Garrick D, Gibbons RJ, Higgs DR, Cristea IM, Urnov FD, Zheng D, Allis CD.

Cell. 2010 Mar 5;140(5):678-91. doi: 10.1016/j.cell.2010.01.003.

16.

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.

Nat Biotechnol. 2009 Sep;27(9):851-7. doi: 10.1038/nbt.1562. Epub 2009 Aug 13.

17.

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.

Nature. 2009 May 21;459(7245):437-41. doi: 10.1038/nature07992. Epub 2009 Apr 29.

PMID:
19404259
18.

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.

Plant Mol Biol. 2009 Apr;69(6):699-709. doi: 10.1007/s11103-008-9449-7. Epub 2008 Dec 27.

PMID:
19112554
19.

Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases.

Moehle EA, Rock JM, Lee YL, Jouvenot Y, DeKelver RC, Gregory PD, Urnov FD, Holmes MC.

Proc Natl Acad Sci U S A. 2007 Feb 27;104(9):3055-60. Epub 2007 Feb 20. Erratum in: Proc Natl Acad Sci U S A. 2007 Apr 3;104(14):6090. Moehle, E A [corrected to Moehle, Erica A]; Rock, J M [corrected to Rock, Jeremy M]; Lee, Y L [corrected to Lee, Ya-Li]; Jouvenot, Y [corrected to Jouvenot, Yann]; Dekelver, R C [corrected to DeKelver, Russell C]; Gregory, P D [corrected to Gregory, Philip.

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