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Page 1
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression.
Handyside B, Zhang L, Yates B, Xie L, Ismail AM, Murphy R, Baridon B, Su C, Bouwman T, Mangini L, Tahquechi J, Salcido S, Minto WC, Keenan WT, Ntai I, Sihn CR, Bullens S, Bunting S, Fong S. Handyside B, et al. Among authors: sihn cr. Hum Gene Ther. 2024 Jan;35(1-2):36-47. doi: 10.1089/hum.2023.065. Hum Gene Ther. 2024. PMID: 38126359 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: sihn cr. 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. Among authors: sihn cr. 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.
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: sihn cr. 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.
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: sihn cr. 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.
Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells.
Handyside B, Ismail AM, Zhang L, Yates B, Xie L, Sihn CR, Murphy R, Bouwman T, Kim CK, De Angelis R, Karim OA, McIntosh NL, Doss MX, Shroff S, Pungor E, Bhat VS, Bullens S, Bunting S, Fong S. Handyside B, et al. Among authors: sihn cr. Mol Ther. 2022 Dec 7;30(12):3570-3586. doi: 10.1016/j.ymthe.2022.11.001. Epub 2022 Nov 7. Mol Ther. 2022. PMID: 36348622 Free PMC article.
31 results