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Strategy to detect pre-existing immunity to AAV gene therapy.
Falese L, Sandza K, Yates B, Triffault S, Gangar S, Long B, Tsuruda L, Carter B, Vettermann C, Zoog SJ, Fong S. Falese L, et al. Among authors: fong s. Gene Ther. 2017 Dec;24(12):768-778. doi: 10.1038/gt.2017.95. Epub 2017 Nov 6. Gene Ther. 2017. PMID: 29106404 Free PMC article.
Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice.
Bunting S, Zhang L, Xie L, Bullens S, Mahimkar R, Fong S, Sandza K, Harmon D, Yates B, Handyside B, Sihn CR, Galicia N, Tsuruda L, O'Neill CA, Bagri A, Colosi P, Long S, Vehar G, Carter B. Bunting S, et al. Among authors: fong s. Mol Ther. 2018 Feb 7;26(2):496-509. doi: 10.1016/j.ymthe.2017.12.009. Epub 2017 Dec 14. Mol Ther. 2018. PMID: 29292164 Free PMC article.
Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter.
Fong S, Handyside B, Sihn CR, Liu S, Zhang L, Xie L, Murphy R, Galicia N, Yates B, Minto WC, Vitelli C, Harmon D, Ru Y, Yu GK, Escher C, Vowinckel J, Woloszynek J, Akeefe H, Mahimkar R, Bullens S, Bunting S. Fong S, et al. Mol Ther Methods Clin Dev. 2020 Jul 9;18:620-630. doi: 10.1016/j.omtm.2020.07.005. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775496 Free PMC article.
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers.
Sihn CR, Handyside B, Liu S, Zhang L, Murphy R, Yates B, Xie L, Torres R, Russell CB, O'Neill CA, Pungor E, Bunting S, Fong S. Sihn CR, et al. Among authors: fong s. Mol Ther Methods Clin Dev. 2021 Dec 21;24:142-153. doi: 10.1016/j.omtm.2021.12.004. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 35036471 Free PMC article.
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood.
Zhang L, Yates B, Murphy R, Liu S, Xie L, Handyside B, Sihn CR, Bouwman T, Galicia N, Tan D, Fonck C, Arens J, Clark A, Zhang W, Chandra S, Srimani J, Holcomb J, Van Tuyl A, Henshaw J, Vettermann C, Siso S, Su C, Bullens S, Bunting S, O'Neill C, Fong S. Zhang L, et al. Among authors: fong s. Mol Ther Methods Clin Dev. 2022 Aug 13;26:519-531. doi: 10.1016/j.omtm.2022.08.002. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 36092364 Free PMC article.
Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action.
Liu S, Razon L, Ritchie O, Sihn CR, Handyside B, Berguig G, Woloszynek J, Zhang L, Batty P, Lillicrap D, Agrawal V, Cortesio C, Gebretsadik K, Akeefe H, Colosi P, Kim B, Bunting S, Fong S. Liu S, et al. Among authors: fong s. Mol Ther Methods Clin Dev. 2022 May 29;26:61-71. doi: 10.1016/j.omtm.2022.05.008. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 35782594 Free PMC article.
Gene therapy for hemophilia.
Lenting PJ, Fong S. Lenting PJ, et al. Among authors: fong s. Blood Adv. 2024 Dec 10;8(23):6081. doi: 10.1182/bloodadvances.2024013864. Blood Adv. 2024. PMID: 39378582 Free PMC article. No abstract available.
1,070 results