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

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

Content Provider	MDPI
Author	Sonoda, Hiroyuki Takahashi, Kenichi Minami, Kohtaro Hirato, Toru Yamamoto, Tatsuyoshi So, Sairei Tanizawa, Kazunori Schmidt, Mathias Sato, Yuji
Copyright Year	2022
Description	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 impervious to the therapeutic benefits of ERT, although ERT via intrathecal and intracerebroventricular routes can be used for some neuronopathic LSDs (in particular, mucopolysaccharidoses). However, the considerable practical issues involved make these routes unsuitable for long-term treatment. Efforts have been made to modify enzymes (e.g., by fusing them with antibodies against innate receptors on the cerebrovascular endothelium) so that they can cross the BBB via receptor-mediated transcytosis (RMT) and address neuronopathy in the CNS. This review summarizes the various scientific and technological challenges of applying RMT to the development of safe and effective enzyme therapeutics for neuronopathic mucopolysaccharidoses; it then discusses the translational and methodological issues surrounding preclinical and clinical evaluation to establish RMT-applied ERT.
Starting Page	1240
e-ISSN	19994923
DOI	10.3390/pharmaceutics14061240
Journal	Pharmaceutics
Issue Number	6
Volume Number	14
Language	English
Publisher	MDPI
Publisher Date	2022-06-11
Access Restriction	Open
Subject Keyword	Pharmaceutics Lysosomal Storage Disease Neuronopathic Mucopolysaccharidosis Blood–brain Barrier Neurodegeneration Enzyme Replacement Therapy Receptor-mediated Transcytosis Transferrin Receptor Insulin Receptor
Content Type	Text
Resource Type	Article

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