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AAV-mediated gene therapy for Sialidosis.
van de Vlekkert D, Hu H, Fremuth LE, Brown SA, Weesner JA, Gomero E, Campos Y, d'Azzo A. van de Vlekkert D, et al. Among authors: fremuth le. bioRxiv [Preprint]. 2023 Nov 13:2023.11.10.566667. doi: 10.1101/2023.11.10.566667. bioRxiv. 2023. PMID: 38014061 Free PMC article. Preprint.
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: fremuth le. J Clin Med. 2020 Mar 4;9(3):695. doi: 10.3390/jcm9030695. J Clin Med. 2020. PMID: 32143456 Free PMC article.
AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study.
Hu H, Mosca R, Gomero E, van de Vlekkert D, Campos Y, Fremuth LE, Brown SA, Weesner JA, Annunziata I, d'Azzo A. Hu H, et al. Among authors: fremuth le. Mol Ther Methods Clin Dev. 2021 Oct 28;23:644-658. doi: 10.1016/j.omtm.2021.10.007. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34901309 Free PMC article.