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First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. Harmatz P, et al. Among authors: cao l. Mol Ther. 2022 Dec 7;30(12):3587-3600. doi: 10.1016/j.ymthe.2022.10.010. Epub 2022 Oct 25. Mol Ther. 2022. PMID: 36299240 Free PMC article.
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Yasuda M, Huston MW, Pagant S, Gan L, St Martin S, Sproul S, Richards D, Ballaron S, Hettini K, Ledeboer A, Falese L, Cao L, Lu Y, Holmes MC, Meyer K, Desnick RJ, Wechsler T. Yasuda M, et al. Among authors: cao l. Mol Ther Methods Clin Dev. 2020 Jul 9;18:607-619. doi: 10.1016/j.omtm.2020.07.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775495 Free PMC article.
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Leavitt AD, Konkle BA, Stine KC, Visweshwar N, Harrington TJ, Giermasz A, Arkin S, Fang A, Plonski F, Yver A, Ganne F, Agathon D, Resa MLA, Tseng LJ, Di Russo G, Cockroft BM, Cao L, Rupon J. Leavitt AD, et al. Among authors: cao l. Blood. 2024 Feb 29;143(9):796-806. doi: 10.1182/blood.2022018971. Blood. 2024. PMID: 37871576 Free PMC article. Clinical Trial.
Essential role of glycoprotein Ibα in platelet activation.
Yan R, Xia Y, Zhou K, Liu J, Sun Y, He C, Ge X, Yang M, Sun C, Yuan L, Li S, Yang B, Meng F, Cao L, Ruan C, Dai K. Yan R, et al. Among authors: cao l. Blood Adv. 2024 May 3:bloodadvances.2023012308. doi: 10.1182/bloodadvances.2023012308. Online ahead of print. Blood Adv. 2024. PMID: 38701351
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