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Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I.
Mosca R, van de Vlekkert D, Campos Y, Fremuth LE, Cadaoas J, Koppaka V, Kakkis E, Tifft C, Toro C, Allievi S, Gellera C, Canafoglia L, Visser G, Annunziata I, d'Azzo A. Mosca R, et al. Among authors: kakkis e. J Clin Med. 2020 Mar 4;9(3):695. doi: 10.3390/jcm9030695. J Clin Med. 2020. PMID: 32143456 Free PMC article.
Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept.
Cadaoas J, Hu H, Boyle G, Gomero E, Mosca R, Jayashankar K, Machado M, Cullen S, Guzman B, van de Vlekkert D, Annunziata I, Vellard M, Kakkis E, Koppaka V, d'Azzo A. Cadaoas J, et al. Among authors: kakkis e. Mol Ther Methods Clin Dev. 2020 Nov 20;20:191-203. doi: 10.1016/j.omtm.2020.11.012. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33426146 Free PMC article.
Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.
Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA. Wang L, et al. Among authors: kakkis e. Mol Ther Methods Clin Dev. 2022 Jan 19;24:292-305. doi: 10.1016/j.omtm.2022.01.007. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2022. PMID: 35211641 Free PMC article.
71 results