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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: zhou s. 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.
In vivo genome editing restores haemostasis in a mouse model of haemophilia.
Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, Rebar EJ, Bushman FD, Gregory PD, Holmes MC, High KA. Li H, et al. Among authors: zhou s. Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177. Nature. 2011. PMID: 21706032 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: zhou s. 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.
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA. Arruda VR, et al. Among authors: zhou s. Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24. Blood. 2010. PMID: 20335222 Free PMC article.
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.
McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC. McIntosh J, et al. Among authors: zhou sz, zhou j. Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20. Blood. 2013. PMID: 23426947 Free PMC article.
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