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

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 generally inef...

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Detalhes bibliográficos
Main Authors: Janson, Christopher G., Romanova, Liudmila G., Leone, Paola, Nan, Zhenhong, Belur, Lalitha, McIvor, R. Scott, Low, Walter C.
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4116107/
https://ncbi.nlm.nih.gov/pubmed/24077583
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1227/NEU.0000000000000157
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