Lentiviral gene therapy for mucopolysaccharidosis II with tagged iduronate 2-Sulfatase prevents life-threatening pathology in peripheral tissues but fails to correct cartilage von Catalano, F., Vlaar, E.C., Dammou, Z., Katsavelis, D., Huizer, T.F., Zundo, G., Hoogeveen-Westerveld, M., Oussoren, E., Hout, H.J.M.P. van den, Schaaf, G., Pike-Overzet, K., Staal, F.J.T., Ploeg, A.T. van der, Pijnappel, W.W.M.P.

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Tagged IDS causes efficient and engraftment- independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II von Catalano, F., Vlaar, E.C., Katsavelis, D., Dammou, Z., Huizer, T.F., Bosch, J.C.V., Hoogeveen-Westerveld, M., Hout, H.J.M.P. van den, Oussoren, E., Ruijter, G.J.G., Schaaf, G., Pike-Overzet, K., Staal, F.J.T., Ploeg, A.T. van der, Pijnappel, W.W.M.P.

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