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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Опубликовано в: :Front Mol Neurosci
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: Frontiers Media S.A. 2021
Предметы:
Neuroscience
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC8141728/
https://ncbi.nlm.nih.gov/pubmed/34040503
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fnmol.2021.618360
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