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Lentiviral vectors can be used for full-length dystrophin gene therapy.
Counsell JR, Asgarian Z, Meng J, Ferrer V, Vink CA, Howe SJ, Waddington SN, Thrasher AJ, Muntoni F, Morgan JE, Danos O. Counsell JR, et al. Among authors: waddington sn. Sci Rep. 2017 Mar 6;7(1):79. doi: 10.1038/s41598-017-00152-5. Sci Rep. 2017. PMID: 28250438 Free PMC article.
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.
Waddington SN, Nivsarkar MS, Mistry AR, Buckley SM, Kemball-Cook G, Mosley KL, Mitrophanous K, Radcliffe P, Holder MV, Brittan M, Georgiadis A, Al-Allaf F, Bigger BW, Gregory LG, Cook HT, Ali RR, Thrasher A, Tuddenham EG, Themis M, Coutelle C. Waddington SN, et al. Blood. 2004 Nov 1;104(9):2714-21. doi: 10.1182/blood-2004-02-0627. Epub 2004 Jul 1. Blood. 2004. PMID: 15231566 Free article.
Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice.
Themis M, Waddington SN, Schmidt M, von Kalle C, Wang Y, Al-Allaf F, Gregory LG, Nivsarkar M, Themis M, Holder MV, Buckley SM, Dighe N, Ruthe AT, Mistry A, Bigger B, Rahim A, Nguyen TH, Trono D, Thrasher AJ, Coutelle C. Themis M, et al. Among authors: waddington sn. Mol Ther. 2005 Oct;12(4):763-71. doi: 10.1016/j.ymthe.2005.07.358. Mol Ther. 2005. PMID: 16084128 Free article.
Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX.
Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M. Bigger BW, et al. Among authors: waddington sn. Gene Ther. 2006 Jan;13(2):117-26. doi: 10.1038/sj.gt.3302638. Gene Ther. 2006. PMID: 16163377
Stable gene transfer to muscle using non-integrating lentiviral vectors.
Apolonia L, Waddington SN, Fernandes C, Ward NJ, Bouma G, Blundell MP, Thrasher AJ, Collins MK, Philpott NJ. Apolonia L, et al. Among authors: waddington sn. Mol Ther. 2007 Nov;15(11):1947-54. doi: 10.1038/sj.mt.6300281. Epub 2007 Aug 14. Mol Ther. 2007. PMID: 17700544 Free article.
172 results