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A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
Nguyen GN, Everett JK, Kafle S, Roche AM, Raymond HE, Leiby J, Wood C, Assenmacher CA, Merricks EP, Long CT, Kazazian HH, Nichols TC, Bushman FD, Sabatino DE. Nguyen GN, et al. Among authors: sabatino de. Nat Biotechnol. 2021 Jan;39(1):47-55. doi: 10.1038/s41587-020-0741-7. Epub 2020 Nov 16. Nat Biotechnol. 2021. PMID: 33199875 Free PMC article.
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. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
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: sabatino de. Nat Med. 2007 Apr;13(4):419-22. doi: 10.1038/nm1549. Epub 2007 Mar 18. Nat Med. 2007. PMID: 17369837
Animal models of hemophilia.
Sabatino DE, Nichols TC, Merricks E, Bellinger DA, Herzog RW, Monahan PE. Sabatino DE, et al. Prog Mol Biol Transl Sci. 2012;105:151-209. doi: 10.1016/B978-0-12-394596-9.00006-8. Prog Mol Biol Transl Sci. 2012. PMID: 22137432 Free PMC article. Review.
Muscle Gene Therapy for Hemophilia.
Sabatino DE, Arruda VR. Sabatino DE, et al. J Genet Syndr Gene Ther. 2012 May 7;Suppl 1:S1-010. doi: 10.4172/2157-7412.s1-010. J Genet Syndr Gene Ther. 2012. PMID: 24883231 Free PMC article.
44 results