Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: a gene therapy for Fabry disease

Author(s):
Liefhebber, J.M.P.; Brasser, G.; Spronck, E.A.; Ottenhoff, R.; Paerels, L.; Guimaraes Da Lomba Ferraz, M.J.; Schwarz, L.K.; Efthymiopoulou, N.; Kuo, C.-L.; Montenegro-Miranda, P.S.; Evers, M.M.; Aerts, J.M.F.G. (0000-0001-8168-2565); Liu, Y.P.
Published
2024
Publication type
Article / Letter to editor
Date issued
2024-12-12
Language
English
Is part of
Molecular Therapy: Methods and Clinical Development (2024), No. 4, 101375
hdl
1887/4178604
doi
10.1016/j.omtm.2024.101375