Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II
Titel:
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II
Auteur:
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