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IL-15 blockade and rapamycin rescue multifactorial loss of factor VIII from AAV-transduced hepatocytes in hemophilia A mice.
Butterfield JSS, Yamada K, Bertolini TB, Syed F, Kumar SRP, Li X, Arisa S, Piñeros AR, Tapia A, Rogers CA, Li N, Rana J, Biswas M, Terhorst C, Kaufman RJ, de Jong YP, Herzog RW. Butterfield JSS, et al. Among authors: li x, li n. Mol Ther. 2022 Dec 7;30(12):3552-3569. doi: 10.1016/j.ymthe.2022.07.005. Epub 2022 Jul 12. Mol Ther. 2022. PMID: 35821634 Free PMC article.
Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies.
Baik AD, Calafati P, Zhang X, Aaron NA, Mehra A, Moller-Tank S, Miloscio L, Praggastis M, Giovannone N, Pan C, Tang Y, Bridges S, Mujica A, Barbounis P, Yanolatos J, Gale N, Li N, Kyratsous CA, Schoenherr CJ, Murphy AJ, Economides AN, Cygnar KD. Baik AD, et al. Among authors: li n. Mol Ther. 2021 Dec 1;29(12):3512-3524. doi: 10.1016/j.ymthe.2021.08.020. Epub 2021 Aug 14. Mol Ther. 2021. PMID: 34400331 Free PMC article.
Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity.
Biswas M, Marsic D, Li N, Zou C, Gonzalez-Aseguinolaza G, Zolotukhin I, Kumar SRP, Rana J, Butterfield JSS, Kondratov O, de Jong YP, Herzog RW, Zolotukhin S. Biswas M, et al. Among authors: li n. Mol Ther Methods Clin Dev. 2020 Oct 4;19:347-361. doi: 10.1016/j.omtm.2020.09.019. eCollection 2020 Dec 11. Mol Ther Methods Clin Dev. 2020. PMID: 33145371 Free PMC article.
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