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

Mucopolysaccharidosis type II (OMIM 309900) is a lysosomal storage disorder caused by iduronate 2-sulfatase (IDS) deficiency and accumulation of glycosaminoglycans, leading to progressive neurodegeneration. As intravenously infused enzyme replacement therapy cannot cross the blood-brain barrier (BBB...

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Главные авторы:	Fabio Catalano, Eva C. Vlaar, Drosos Katsavelis, Zina Dammou, Tessa F. Huizer, Jeroen C. van den Bosch, Marianne Hoogeveen-Westerveld, Hannerieke J.M.P. van den Hout, Esmeralda Oussoren, George J.G. Ruijter, Gerben Schaaf, Karin Pike-Overzet, Frank J.T. Staal, Ans T. van der Ploeg, W.W.M. Pim Pijnappel
Формат:	Artigo
Язык:	Inglês
Опубликовано:	Elsevier 2023-12-01
Серии:	Molecular Therapy: Methods & Clinical Development
Предметы:	Hunter disease mucopolysaccharidosis type II lentiviral gene therapy IDS IGF2 ApoE2
Online-ссылка:	http://www.sciencedirect.com/science/article/pii/S2329050123001882
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