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.
Mol Pharm. 2023 Nov 6;20(11):5901-5909. doi: 10.1021/acs.molpharmaceut.3c00736. Epub 2023 Oct 20.
PMID:37860991
Ready-to-use iPSC-derived microglia progenitors for the treatment of CNS disease in mouse models of neuropathic mucopolysaccharidoses.
Douvaras P, Buenaventura DF, Sun B, Lepack A, Baker E, Simpson E, Ebel M, Lallos G, LoSchiavo D, Stitt N, Adams N, McAuliffe C, Forton-Juarez A, Kosmyna B, Pereira E, Burnett B, Dilworth D, Fisher S, Wang J, Tonge P, Tomishima M, Paladini C, Wilkinson D, Soh CL, Srinivas M, Patsch C, Irion S.