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

Imakiire, A; Morimoto, H; Suzuki, H; Masuda, T; Yoden, E; Inoue, A; Morioka, H; Konaka, T; Mori, A; Shirasaka, R; Kato, R; Hirato, T; Sonoda, H; Minami, K

Minami, K (通讯作者),JCR Pharmaceut, Res Div, Kobe 6512241, Japan.

MOLECULAR PHARMACEUTICS, 2023; 20 (11): 5901

Abstract

Mucopolysaccharidoses (MPSs) make up a group of lysosomal storage diseases characterized by the aberrant accumulation of glycosaminoglycans throughout......

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