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Page 1
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. Ou L, et al. Among authors: holmes mc. Mol Ther. 2019 Jan 2;27(1):178-187. doi: 10.1016/j.ymthe.2018.10.018. Epub 2018 Nov 1. Mol Ther. 2019. PMID: 30528089 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: holmes mc. 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.
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: holmes mc. 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.
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: holmes mc. 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.
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Yasuda M, Huston MW, Pagant S, Gan L, St Martin S, Sproul S, Richards D, Ballaron S, Hettini K, Ledeboer A, Falese L, Cao L, Lu Y, Holmes MC, Meyer K, Desnick RJ, Wechsler T. Yasuda M, et al. Among authors: holmes mc. Mol Ther Methods Clin Dev. 2020 Jul 9;18:607-619. doi: 10.1016/j.omtm.2020.07.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775495 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: holmes mc. 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.
Robust ZFN-mediated genome editing in adult hemophilic mice.
Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA. Anguela XM, et al. Among authors: holmes mc. Blood. 2013 Nov 7;122(19):3283-7. doi: 10.1182/blood-2013-04-497354. Epub 2013 Oct 1. Blood. 2013. PMID: 24085764 Free PMC article.
Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells.
DiGiusto DL, Cannon PM, Holmes MC, Li L, Rao A, Wang J, Lee G, Gregory PD, Kim KA, Hayward SB, Meyer K, Exline C, Lopez E, Henley J, Gonzalez N, Bedell V, Stan R, Zaia JA. DiGiusto DL, et al. Among authors: holmes mc. Mol Ther Methods Clin Dev. 2016 Nov 9;3:16067. doi: 10.1038/mtm.2016.67. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27900346 Free PMC article.
230 results