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Page 1
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.
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.
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.
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.
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 article.
Targeting oxidized LDL improves insulin sensitivity and immune cell function in obese Rhesus macaques.
Li S, Kievit P, Robertson AK, Kolumam G, Li X, von Wachenfeldt K, Valfridsson C, Bullens S, Messaoudi I, Bader L, Cowan KJ, Kamath A, van Bruggen N, Bunting S, Frendéus B, Grove KL. Li S, et al. Among authors: bullens s. Mol Metab. 2013 Jun 11;2(3):256-69. doi: 10.1016/j.molmet.2013.06.001. eCollection 2013. Mol Metab. 2013. PMID: 24049738 Free PMC article.
Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice.
Chen JC, Luu AR, Wise N, Angelis R, Agrawal V, Mangini L, Vincelette J, Handyside B, Sterling H, Lo MJ, Wong H, Galicia N, Pacheco G, Van Vleet J, Giaramita A, Fong S, Roy SM, Hague C, Lawrence R, Bullens S, Christianson TM, d'Azzo A, Crawford BE, Bunting S, LeBowitz JH, Yogalingam G. Chen JC, et al. Among authors: bullens s. J Biol Chem. 2020 Sep 25;295(39):13532-13555. doi: 10.1074/jbc.RA119.009811. Epub 2019 Sep 3. J Biol Chem. 2020. PMID: 31481471 Free PMC article.
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Kan SH, Aoyagi-Scharber M, Le SQ, Vincelette J, Ohmi K, Bullens S, Wendt DJ, Christianson TM, Tiger PM, Brown JR, Lawrence R, Yip BK, Holtzinger J, Bagri A, Crippen-Harmon D, Vondrak KN, Chen Z, Hague CM, Woloszynek JC, Cheung DS, Webster KA, Adintori EG, Lo MJ, Wong W, Fitzpatrick PA, LeBowitz JH, Crawford BE, Bunting S, Dickson PI, Neufeld EF. Kan SH, et al. Among authors: bullens s. Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14870-5. doi: 10.1073/pnas.1416660111. Epub 2014 Sep 29. Proc Natl Acad Sci U S A. 2014. PMID: 25267636 Free PMC article.
29 results