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Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease.
Liefhebber JMP, Brasser G, Spronck EA, Ottenhoff R, Paerels L, Ferraz MJ, Schwarz LK, Efthymiopoulou N, Kuo CL, Montenegro-Miranda PS, Evers MM, Aerts JMFG, Liu YP. Liefhebber JMP, et al. Among authors: aerts jmfg. Mol Ther Methods Clin Dev. 2024 Nov 12;32(4):101375. doi: 10.1016/j.omtm.2024.101375. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39687734 Free PMC article.
461 results