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Page 1
Optimizing liver health before and after gene therapy for hemophilia A.
Ragni MV, Mead H, de Jong YP, Kaczmarek R, Leavitt AD, Long B, Nugent DJ, Sabatino DE, Fong S, von Drygalski A, Walsh CE, Luxon BA. Ragni MV, et al. Among authors: fong s. Blood Adv. 2024 Oct 8;8(19):5203-5212. doi: 10.1182/bloodadvances.2024013059. Blood Adv. 2024. PMID: 38843379 Free PMC article. Review.
Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model.
Lopez ME, Wendt D, Lawrence R, Gong K, Ong H, Yip B, Chen J, Mangini L, Handyside B, Giaramita A, Lamichhane A, Lo M, Agrawal V, Van Vleet J, Abolhesn A, Felix JB, Villalpando I, Bhat V, De Angelis R, Ru Y, Khan A, Fong S, Christianson T, Bullens S, Crawford BE, Bunting S, Aoyagi-Scharber M. Lopez ME, et al. Among authors: fong s. PLoS One. 2025 Jan 3;20(1):e0315005. doi: 10.1371/journal.pone.0315005. eCollection 2025. PLoS One. 2025. PMID: 39752451 Free PMC article.
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: fong s. 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.
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: fong s. 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.
Valoctocogene roxaparvovec gene transfer in participants with HIV.
Ragni MV, Majerus E, Fong S, Yates B, Scheeler S, Razon L, Yu H, Reddy DB, Robinson TM. Ragni MV, et al. Among authors: fong s. Blood Adv. 2023 Apr 25;7(8):1525-1530. doi: 10.1182/bloodadvances.2022008948. Blood Adv. 2023. PMID: 36696462 Free PMC article. No abstract available.
1,067 results