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

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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Bibliographic Details
Main Authors: Lalitha R. Belur, Megan Romero, Junggu Lee, Kelly M. Podetz-Pedersen, Zhenhong Nan, Maureen S. Riedl, Lucy Vulchanova, Kelley F. Kitto, Carolyn A. Fairbanks, Karen F. Kozarsky, Paul J. Orchard, William H. Frey, Walter C. Low, R. Scott McIvor
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-05-01
Series:Frontiers in Molecular Neuroscience
Subjects:
MPS I
IDUA
AAV9
gene therapy
intracerebroventricular administration
intrathecal injection
Online Access:https://www.frontiersin.org/articles/10.3389/fnmol.2021.618360/full

Internet

https://www.frontiersin.org/articles/10.3389/fnmol.2021.618360/full

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