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Page 1
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: bullens 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: bullens 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: bullens 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.
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction.
Magat J, Jones S, Baridon B, Agrawal V, Wong H, Giaramita A, Mangini L, Handyside B, Vitelli C, Parker M, Yeung N, Zhou Y, Pungor E, Slabodkin I, Gorostiza O, Aguilera A, Lo MJ, Alcozie S, Christianson TM, Tiger PMN, Vincelette J, Fong S, Gil G, Hague C, Lawrence R, Wendt DJ, Lebowitz JH, Bunting S, Bullens S, Crawford BE, Roy SM, Woloszynek JC. Magat J, et al. Among authors: bullens s. J Biol Chem. 2022 Dec;298(12):102625. doi: 10.1016/j.jbc.2022.102625. Epub 2022 Oct 26. J Biol Chem. 2022. PMID: 36306823 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: bullens 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.
Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency.
Luu AR, Wong C, Agrawal V, Wise N, Handyside B, Lo MJ, Pacheco G, Felix JB, Giaramita A, d'Azzo A, Vincelette J, Bullens S, Bunting S, Christianson TM, Hague CM, LeBowitz JH, Yogalingam G. Luu AR, et al. Among authors: bullens s. J Biol Chem. 2020 Nov 13;295(46):15794. doi: 10.1074/jbc.AAC120.016409. J Biol Chem. 2020. PMID: 33188082 Free PMC article. No abstract available.
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: bullens s. 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.
Intermittent enzyme replacement therapy with recombinant human β-galactosidase prevents neuraminidase 1 deficiency.
Luu AR, Wong C, Agrawal V, Wise N, Handyside B, Lo MJ, Pacheco G, Felix JB, Giaramita A, d'Azzo A, Vincelette J, Bullens S, Bunting S, Christianson TM, Hague CM, LeBowitz JH, Yogalingam G. Luu AR, et al. Among authors: bullens s. J Biol Chem. 2020 Sep 25;295(39):13556-13569. doi: 10.1074/jbc.RA119.010794. Epub 2020 Jul 28. J Biol Chem. 2020. PMID: 32727849 Free PMC article.
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
Grover A, Crippen-Harmon D, Nave L, Vincelette J, Wait JCM, Melton AC, Lawrence R, Brown JR, Webster KA, Yip BK, Baridon B, Vitelli C, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Shaywitz AJ, Crawford BE, Fitzpatrick PA, LeBowitz JH, Bullens S, Aoyagi-Scharber M, Bunting S, O'Neill CA, Pinkstaff J, Bagri A. Grover A, et al. Among authors: bullens s. Drug Deliv Transl Res. 2020 Apr;10(2):425-439. doi: 10.1007/s13346-019-00683-6. Drug Deliv Transl Res. 2020. PMID: 31942701 Free PMC article.
31 results