Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease

Abstract BACKGROUND: Hurler disease (mucopolysaccharidosis type I [MPS-I]) is an inherited metabolic disorder characterized by deficiency of the lysosomal enzyme α-L-iduronidase (IDUA). Currently, the only therapies for MPS-I, enzyme replacement and hematopoietic stem cell transplantation, are gener...

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Veröffentlicht in:	Neurosurgery 2014-01, Vol.74 (1), p.99-111
Hauptverfasser:	Janson, Christopher G., Romanova, Liudmila G., Leone, Paola, Nan, Zhenhong, Belur, Lalitha, McIvor, R. Scott, Low, Walter C.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Blood-brain barrier Dependovirus - genetics Disease Models, Animal Enzymes Gene therapy Genetic Therapy - methods Genetic Vectors Humans Iduronidase - administration & dosage Iduronidase - genetics Injections, Intra-Arterial Injections, Intraventricular Mice Mice, Transgenic Mucopolysaccharidosis I - pathology Neurosurgery Rodents
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