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GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals

Recent studies in non-human model systems have shown therapeutic potential of nucleoside-modified messenger RNA (modRNA) treatments for lysosomal storage diseases. Here, we assessed the efficacy of a modRNA treatment to restore the expression of the galactosidase alpha (GLA), which codes for α-Galac...

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Detalles Bibliográficos
Autores principales:	ter Huurne, Menno, Parker, Benjamin L., Liu, Ning Qing, Qian, Elizabeth Ling, Vivien, Celine, Karavendzas, Kathy, Mills, Richard J., Saville, Jennifer T., Abu-Bonsrah, Dad, Wise, Andrea F., Hudson, James E., Talbot, Andrew S., Finn, Patrick F., Martini, Paolo G.V., Fuller, Maria, Ricardo, Sharon D., Watt, Kevin I., Nicholls, Kathy M., Porrello, Enzo R., Elliott, David A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2023
Materias:	Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10502840/ https://www.ncbi.nlm.nih.gov/pubmed/37607539 http://dx.doi.org/10.1016/j.ajhg.2023.07.013

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