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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Manno CS, et al. Among authors: high ka. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B. Manno CS, et al. Among authors: high ka. Blood. 2003 Apr 15;101(8):2963-72. doi: 10.1182/blood-2002-10-3296. Epub 2002 Dec 19. Blood. 2003. PMID: 12515715 Free article.
CD8(+) T-cell responses to adeno-associated virus capsid in humans.
Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA. Mingozzi F, et al. Among authors: high ka. Nat Med. 2007 Apr;13(4):419-22. doi: 10.1038/nm1549. Epub 2007 Mar 18. Nat Med. 2007. PMID: 17369837
Immune responses to AAV in clinical trials.
Mingozzi F, High KA. Mingozzi F, et al. Among authors: high ka. Curr Gene Ther. 2007 Oct;7(5):316-24. doi: 10.2174/156652307782151425. Curr Gene Ther. 2007. PMID: 17979678 Review.
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.
239 results