Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
| Autor: | 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 |
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| Zdroj: | Human Gene Therapy; Apr2024, Vol. 35 Issue 7/8, p256-268, 13p |
| Databáze: | Complementary Index |
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