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Exploring the Pathophysiologic Cascade Leading to Osteoclastogenic Activation in Gaucher Disease Monocytes Generated via CRISPR/Cas9 Technology.
Int J Mol Sci. 2023 Jul 7;24(13):11204. doi: 10.3390/ijms241311204.
Int J Mol Sci. 2023.
PMID: 37446383
Free PMC article.
In vitro and in vivo effects of Ambroxol chaperone therapy in two Italian patients affected by neuronopathic Gaucher disease and epilepsy.
Ciana G, Dardis A, Pavan E, Da Riol RM, Biasizzo J, Ferino D, Zanatta M, Boni A, Antonini L, Crichiutti G, Bembi B.
Ciana G, et al. Among authors: ferino d.
Mol Genet Metab Rep. 2020 Nov 21;25:100678. doi: 10.1016/j.ymgmr.2020.100678. eCollection 2020 Dec.
Mol Genet Metab Rep. 2020.
PMID: 33294373
Free PMC article.
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