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Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The two current treatments [hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT)], are insufficiently effective in addressin...

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Detalles Bibliográficos
Autores principales:	Belur, Lalitha R., Romero, Megan, Lee, Junggu, Podetz-Pedersen, Kelly M., Nan, Zhenhong, Riedl, Maureen S., Vulchanova, Lucy, Kitto, Kelley F., Fairbanks, Carolyn A., Kozarsky, Karen F., Orchard, Paul J., Frey, William H., Low, Walter C., McIvor, R. Scott
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2021
Materias:	Neuroscience
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8141728/ https://www.ncbi.nlm.nih.gov/pubmed/34040503 http://dx.doi.org/10.3389/fnmol.2021.618360

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