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Page 1
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: bunting 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.
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: bunting 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.
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: bunting 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.
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: bunting 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.
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. 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.
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
Aoyagi-Scharber M, Crippen-Harmon D, Lawrence R, Vincelette J, Yogalingam G, Prill H, Yip BK, Baridon B, Vitelli C, Lee A, Gorostiza O, Adintori EG, Minto WC, Van Vleet JL, Yates B, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Fitzpatrick PA, LeBowitz JH, Bullens S, Crawford BE, Bunting S. Aoyagi-Scharber M, et al. Among authors: bunting s. Mol Ther Methods Clin Dev. 2017 Jun 6;6:43-53. doi: 10.1016/j.omtm.2017.05.009. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28664165 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: bunting 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.
Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis.
Vuillemenot BR, Katz ML, Coates JR, Kennedy D, Tiger P, Kanazono S, Lobel P, Sohar I, Xu S, Cahayag R, Keve S, Koren E, Bunting S, Tsuruda LS, O'Neill CA. Vuillemenot BR, et al. Among authors: bunting s. Mol Genet Metab. 2011 Nov;104(3):325-37. doi: 10.1016/j.ymgme.2011.06.018. Epub 2011 Jun 30. Mol Genet Metab. 2011. PMID: 21784683
290 results