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Page 1
Dystrophin immunity in Duchenne's muscular dystrophy.
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. Mendell JR, et al. Among authors: mcphee sw. N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228. N Engl J Med. 2010. PMID: 20925545 Free PMC article. Clinical Trial.
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS, Li J, Wang B, Monahan PE, Rabinowitz JE, Grieger JC, Govindasamy L, Agbandje-McKenna M, Xiao X, Samulski RJ. Bowles DE, et al. Among authors: mcphee sw. Mol Ther. 2012 Feb;20(2):443-55. doi: 10.1038/mt.2011.237. Epub 2011 Nov 8. Mol Ther. 2012. PMID: 22068425 Free PMC article. Clinical Trial.
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.
Monahan PE, Sun J, Gui T, Hu G, Hannah WB, Wichlan DG, Wu Z, Grieger JC, Li C, Suwanmanee T, Stafford DW, Booth CJ, Samulski JJ, Kafri T, McPhee SW, Samulski RJ. Monahan PE, et al. Among authors: mcphee sw. Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21. Hum Gene Ther. 2015. PMID: 25419787 Free PMC article.
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Konkle BA, Walsh CE, Escobar MA, Josephson NC, Young G, von Drygalski A, McPhee SWJ, Samulski RJ, Bilic I, de la Rosa M, Reipert BM, Rottensteiner H, Scheiflinger F, Chapin JC, Ewenstein B, Monahan PE. Konkle BA, et al. Among authors: mcphee swj. Blood. 2021 Feb 11;137(6):763-774. doi: 10.1182/blood.2019004625. Blood. 2021. PMID: 33067633 Free PMC article. Clinical Trial.
Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.
Ishikawa K, Fish KM, Tilemann L, Rapti K, Aguero J, Santos-Gallego CG, Lee A, Karakikes I, Xie C, Akar FG, Shimada YJ, Gwathmey JK, Asokan A, McPhee S, Samulski J, Samulski RJ, Sigg DC, Weber T, Kranias EG, Hajjar RJ. Ishikawa K, et al. Mol Ther. 2014 Dec;22(12):2038-2045. doi: 10.1038/mt.2014.127. Epub 2014 Jul 15. Mol Ther. 2014. PMID: 25023328 Free PMC article.
Gene therapy for cardiomyocytes, a heart beat away.
McPhee SW, Samulski RJ. McPhee SW, et al. Gene Ther. 2009 Jun;16(6):707-8. doi: 10.1038/gt.2009.40. Epub 2009 Apr 30. Gene Ther. 2009. PMID: 19404321 No abstract available.
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