Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II
Catalano, Fabio, Vlaar, Eva C., Katsavelis, Drosos, Dammou, Zina, Huizer, Tessa F., van den Bosch, Jeroen C., Hoogeveen-Westerveld, Marianne, van den Hout, Hannerieke J.M.P., Oussoren, Esmeralda, Ruijter, George J.G., Schaaf, Gerben, Pike-Overzet, Karin, Staal, Frank J.T., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Published in Molecular therapy. Methods & clinical development (14.12.2023)
Published in Molecular therapy. Methods & clinical development (14.12.2023)
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Journal Article
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage
Catalano, Fabio, Vlaar, Eva C, Dammou, Zina, Katsavelis, Drosos, Huizer, Tessa F, Zundo, Giacomo, Hoogeveen-Westerveld, Marianne, Oussoren, Esmeralda, van den Hout, Hannerieke J M P, Schaaf, Gerben, Pike-Overzet, Karin, Staal, Frank J T, van der Ploeg, Ans T, Pijnappel, W W M Pim
Published in Human gene therapy (01.04.2024)
Published in Human gene therapy (01.04.2024)
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Journal Article