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Page 1
AAV-mediated gene therapy for Sialidosis.
bioRxiv [Preprint]. 2023 Nov 13:2023.11.10.566667. doi: 10.1101/2023.11.10.566667.
bioRxiv. 2023.
PMID: 38014061
Free PMC article.
Preprint.
Altered GM1 catabolism affects NMDAR-mediated Ca 2+ signaling at ER-PM junctions and increases synaptic spine formation.
Weesner JA, Annunziata I, van de Vlekkert D, Robinson CG, Campos Y, Mishra A, Fremuth LE, Gomero E, Hu H, d'Azzo A.
Weesner JA, et al. Among authors: fremuth le.
bioRxiv [Preprint]. 2023 Jul 11:2023.07.10.548446. doi: 10.1101/2023.07.10.548446.
bioRxiv. 2023.
PMID: 37503265
Free PMC article.
Preprint.
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Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis.
Weesner JA, Annunziata I, Yang T, Acosta W, Gomero E, Hu H, van de Vlekkert D, Ayala J, Qiu X, Fremuth LE, Radin DN, Cramer CL, d'Azzo A.
Weesner JA, et al.
Cells. 2022 Aug 19;11(16):2579. doi: 10.3390/cells11162579.
Cells. 2022.
PMID: 36010656
Free PMC article.
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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: fremuth le.
J Clin Med. 2020 Mar 4;9(3):695. doi: 10.3390/jcm9030695.
J Clin Med. 2020.
PMID: 32143456
Free PMC article.
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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.
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Altered GM1 catabolism affects NMDAR-mediated Ca2+ signaling at ER-PM junctions and increases synaptic spine formation in a GM1-gangliosidosis model.
Weesner JA, Annunziata I, van de Vlekkert D, Robinson CG, Campos Y, Mishra A, Fremuth LE, Gomero E, Hu H, d'Azzo A.
Weesner JA, et al. Among authors: fremuth le.
Cell Rep. 2024 Apr 16;43(5):114117. doi: 10.1016/j.celrep.2024.114117. Online ahead of print.
Cell Rep. 2024.
PMID: 38630590
Free article.
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