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Page 1
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. Among authors: samulski j. 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.
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: samulski jj. 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: samulski jj. 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.
Self-complementary AAV2.5-BMP2-coated femoral allografts mediated superior bone healing versus live autografts in mice with equivalent biomechanics to unfractured femur.
Yazici C, Takahata M, Reynolds DG, Xie C, Samulski RJ, Samulski J, Beecham EJ, Gertzman AA, Spilker M, Zhang X, O'Keefe RJ, Awad HA, Schwarz EM. Yazici C, et al. Among authors: samulski j. Mol Ther. 2011 Aug;19(8):1416-25. doi: 10.1038/mt.2010.294. Epub 2011 Jan 4. Mol Ther. 2011. PMID: 21206485 Free PMC article.
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: samulski j. 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.
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Among authors: samulski j. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.
16 results