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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Autors principals:	Hiroyuki Sonoda, Kenichi Takahashi, Kohtaro Minami, Toru Hirato, Tatsuyoshi Yamamoto, Sairei So, Kazunori Tanizawa, Mathias Schmidt, Yuji Sato
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2022-06-01
Col·lecció:	Pharmaceutics
Matèries:	lysosomal storage disease neuronopathic mucopolysaccharidosis blood–brain barrier neurodegeneration enzyme replacement therapy receptor-mediated transcytosis
Accés en línia:	https://www.mdpi.com/1999-4923/14/6/1240
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