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Perspectives on best practices for gene therapy programs.
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK. Cheever TR, et al. Among authors: high ka. Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3. Hum Gene Ther. 2015. PMID: 25654329 Free PMC article.
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.
O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J. O'Reilly M, et al. Among authors: high ka. Hum Gene Ther. 2013 Apr;24(4):355-62. doi: 10.1089/hum.2013.064. Hum Gene Ther. 2013. PMID: 23517518 Free PMC article.
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: high ka. 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.
Progress and prospects: gene therapy clinical trials (part 2).
Aiuti A, Bachoud-Lévi AC, Blesch A, Brenner MK, Cattaneo F, Chiocca EA, Gao G, High KA, Leen AM, Lemoine NR, McNeish IA, Meneguzzi G, Peschanski M, Roncarolo MG, Strayer DS, Tuszynski MH, Waxman DJ, Wilson JM. Aiuti A, et al. Among authors: high ka. Gene Ther. 2007 Nov;14(22):1555-63. doi: 10.1038/sj.gt.3303033. Gene Ther. 2007. PMID: 17984995 Review.
Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines.
Bish LT, Sleeper MM, Reynolds C, Gazzara J, Withnall E, Singletary GE, Buchlis G, Hui D, High KA, Gao G, Wilson JM, Sweeney HL. Bish LT, et al. Among authors: high ka. Hum Gene Ther. 2011 Aug;22(8):969-77. doi: 10.1089/hum.2011.035. Epub 2011 Jun 8. Hum Gene Ther. 2011. PMID: 21542669 Free PMC article.
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.
Haurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, Basner-Tschakarjan E, Arruda VR, Radu A, Franck HG, Wright JF, Zhou S, Stedman HH, Bellinger DA, Nichols TC, High KA. Haurigot V, et al. Among authors: high ka. Mol Ther. 2010 Jul;18(7):1318-29. doi: 10.1038/mt.2010.73. Epub 2010 Apr 27. Mol Ther. 2010. PMID: 20424599 Free PMC article.
Gene therapy for hemophilia: the clot thickens.
High KA. High KA. Hum Gene Ther. 2014 Nov;25(11):915-22. doi: 10.1089/hum.2014.2541. Hum Gene Ther. 2014. PMID: 25397928 Free PMC article. No abstract available.
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.
Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C. Toromanoff A, et al. Among authors: high ka. Mol Ther. 2008 Jul;16(7):1291-9. doi: 10.1038/mt.2008.87. Epub 2008 May 6. Mol Ther. 2008. PMID: 18461055 Free article.
239 results