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Coagulation factor VIII regulates von Willebrand factor homeostasis invivo.
Cao W, Trask AR, Bignotti AI, George LA, Doshi BS, Sabatino DE, Yada N, Zheng L, Camire RM, Zheng XL. Cao W, et al. Among authors: sabatino de. J Thromb Haemost. 2023 Dec;21(12):3477-3489. doi: 10.1016/j.jtha.2023.09.004. Epub 2023 Sep 17. J Thromb Haemost. 2023. PMID: 37726033
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.
Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J; American Society of Gene and Cell Therapy (ASGCT) Working Group on AAV Integration. Sabatino DE, et al. Mol Ther. 2022 Aug 3;30(8):2646-2663. doi: 10.1016/j.ymthe.2022.06.004. Epub 2022 Jun 10. Mol Ther. 2022. PMID: 35690906 Free PMC article. Review.
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.
Elkouby L, Armour SM, Toso R, DiPietro M, Davidson RJ, Nguyen GN, Willet M, Kutza S, Silverberg J, Frick J, Crosariol M, Wang Y, Wang C, High KA, Sabatino DE, Anguela XM. Elkouby L, et al. Among authors: sabatino de. Mol Ther Methods Clin Dev. 2021 Nov 24;24:20-29. doi: 10.1016/j.omtm.2021.11.005. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 34977269 Free PMC article.
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.
Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing.
Zhang X, Zhao W, Nguyen GN, Zhang C, Zeng C, Yan J, Du S, Hou X, Li W, Jiang J, Deng B, McComb DW, Dorkin R, Shah A, Barrera L, Gregoire F, Singh M, Chen D, Sabatino DE, Dong Y. Zhang X, et al. Among authors: sabatino de. Sci Adv. 2020 Aug 21;6(34):eabc2315. doi: 10.1126/sciadv.abc2315. Print 2020 Aug. Sci Adv. 2020. PMID: 32937374 Free PMC article.
40 results