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Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase.
Hordeaux J, Ramezani A, Tuske S, Mehta N, Song C, Lynch A, Lupino K, Chichester JA, Buza EL, Dyer C, Yu H, Bell P, Weimer JM, Do H, Wilson JM. Hordeaux J, et al. Among authors: do h. Front Immunol. 2023 Mar 22;14:1094279. doi: 10.3389/fimmu.2023.1094279. eCollection 2023. Front Immunol. 2023. PMID: 37033976 Free PMC article.
The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease.
Khanna R, Powe AC Jr, Lun Y, Soska R, Feng J, Dhulipala R, Frascella M, Garcia A, Pellegrino LJ, Xu S, Brignol N, Toth MJ, Do HV, Lockhart DJ, Wustman BA, Valenzano KJ. Khanna R, et al. PLoS One. 2014 Jul 18;9(7):e102092. doi: 10.1371/journal.pone.0102092. eCollection 2014. PLoS One. 2014. PMID: 25036864 Free PMC article.
Improved efficacy of a next-generation ERT in murine Pompe disease.
Xu S, Lun Y, Frascella M, Garcia A, Soska R, Nair A, Ponery AS, Schilling A, Feng J, Tuske S, Valle MCD, Martina JA, Ralston E, Gotschall R, Valenzano KJ, Puertollano R, Do HV, Raben N, Khanna R. Xu S, et al. JCI Insight. 2019 Mar 7;4(5):e125358. doi: 10.1172/jci.insight.125358. eCollection 2019 Mar 7. JCI Insight. 2019. PMID: 30843882 Free PMC article.
Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.
McVie-Wylie AJ, Lee KL, Qiu H, Jin X, Do H, Gotschall R, Thurberg BL, Rogers C, Raben N, O'Callaghan M, Canfield W, Andrews L, McPherson JM, Mattaliano RJ. McVie-Wylie AJ, et al. Among authors: do h. Mol Genet Metab. 2008 Aug;94(4):448-455. doi: 10.1016/j.ymgme.2008.04.009. Epub 2008 Jun 5. Mol Genet Metab. 2008. PMID: 18538603 Free PMC article.
1,343 results