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Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice

[Image: see text] Mucopolysaccharidoses (MPSs) make up a group of lysosomal storage diseases characterized by the aberrant accumulation of glycosaminoglycans throughout the body. Patients with MPSs display various signs and symptoms, such as retinopathy, which is also observed in patients with MPS I...

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Detalles Bibliográficos
Autores principales: Imakiire, Atsushi, Morimoto, Hideto, Suzuki, Hidehiko, Masuda, Tomomi, Yoden, Eiji, Inoue, Asuka, Morioka, Hiroki, Konaka, Takashi, Mori, Ayaka, Shirasaka, Ryoji, Kato, Ryo, Hirato, Tohru, Sonoda, Hiroyuki, Minami, Kohtaro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2023
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10630942/
https://www.ncbi.nlm.nih.gov/pubmed/37860991
http://dx.doi.org/10.1021/acs.molpharmaceut.3c00736