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

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Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model by Vanessa Zancanella, Astrid Vallès, Jolanda M.P. Liefhebber, Lieke Paerels, Carlos Vendrell Tornero, Hendrina Wattimury, Tom van der Zon, Kristel van Rooijen, Monika Golinska, Tamar Grevelink, Erich Ehlert, Elsbet Jantine Pieterman, Nanda Keijzer, Hans Marinus Gerardus Princen, Geurt Stokman, Ying Poi Liu

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Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model by Vanessa Zancanella, Astrid Vallès, Jolanda M.P. Liefhebber, Lieke Paerels, Carlos Vendrell Tornero, Hendrina Wattimury, Tom van der Zon, Kristel van Rooijen, Monika Golinska, Tamar Grevelink, Erich Ehlert, Elsbet Jantine Pieterman, Nanda Keijzer, Hans Marinus Gerardus Princen, Geurt Stokman, Ying Poi Liu

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