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Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice

Mucopolysaccharidosis I (MPS I), a lysosomal storage disease caused by dysfunction of α-L-iduronidase (IDUA), is characterized by the deposition of dermatan sulfate (DS) and heparan sulfate (HS) throughout the body, which causes several somatic and central nervous symptoms. Although enzyme-replaceme...

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Detalles Bibliográficos
Autores principales:	Kida, Sachiho, Koshimura, Yuri, Yoden, Eiji, Yoshioka, Aya, Morimoto, Hideto, Imakiire, Atsushi, Tanaka, Noboru, Tanaka, Satowa, Mori, Ayaka, Ito, Jun, Inoue, Asuka, Yamamoto, Ryuji, Minami, Kohtaro, Hirato, Tohru, Takahashi, Kenichi, Sonoda, Hiroyuki
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10220318/ https://www.ncbi.nlm.nih.gov/pubmed/37251981 http://dx.doi.org/10.1016/j.omtm.2023.05.010

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