Efficacy of AAV5-GLA gene therapy in manifest Fabry disease mice von Liefhebber, Jolanda M.P., Brasser, Giso, Ferraz, Maria J., Ottenhoff, Roelof, Efthymiopoulou, Nikoleta, Paerels, Lieke, Pereira, Ines, Sonea, Andra, Allart, Leonie, Schwarz, Lukas K., Squeri, Giorgia, Dobrynin, Greg, Rodriguez, Rosa Crespo, Liu, Ying Poi, Aerts, Johannes M.F.G., Montenegro-Miranda, Paula S.

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Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase A gene therapy for Fabry disease von Liefhebber, Jolanda M.P., Brasser, Giso, Spronck, Elisabeth A., Ottenhoff, Roelof, Paerels, Lieke, Ferraz, Maria J., Schwarz, Lukas K., Efthymiopoulou, Nikoleta, Kuo, Chi-Lin, Montenegro-Miranda, Paula S., Evers, Melvin M., Aerts, Johannes M.F.G., Liu, Ying Poi

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