Treatment of Neuronopathic Mucopolysaccharidoses with Blood–Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis

Treatment of Neuronopathic Mucopolysaccharidoses with Blood–Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis

Enzyme replacement therapy (ERT) has paved the way for treating the somatic symptoms of lysosomal storage diseases (LSDs), but the inability of intravenously administered enzymes to cross the blood–brain barrier (BBB) has left the central nervous system (CNS)-related symptoms of LSDs largely impervi...

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Bibliographic Details
Main Authors: Hiroyuki Sonoda, Kenichi Takahashi, Kohtaro Minami, Toru Hirato, Tatsuyoshi Yamamoto, Sairei So, Kazunori Tanizawa, Mathias Schmidt, Yuji Sato
Format: Article
Language:English
Published: MDPI AG 2022-06-01
Series:Pharmaceutics
Subjects:
lysosomal storage disease
neuronopathic mucopolysaccharidosis
blood–brain barrier
neurodegeneration
enzyme replacement therapy
receptor-mediated transcytosis
Online Access:https://www.mdpi.com/1999-4923/14/6/1240

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https://www.mdpi.com/1999-4923/14/6/1240

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