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

Authors:
Kida S; Koshimura Y; Yoden E; Yoshioka A; Morimoto H and 11 more

Journal:
Mol Ther Methods Clin Dev

Publication Year: 2023

DOI:
10.1016/j.omtm.2023.05.010

PMCID:
PMC10220318

PMID:
37251981

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Journal Information

Full Title: Mol Ther Methods Clin Dev

Abbreviation: Mol Ther Methods Clin Dev

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Medicine, Research & Experimental

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

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Evidence found in paper:

"Declaration of interests S.K., Y.K., E.Y., A.Y., H.M., A. Imakiire, N.T., S.T., A.M., J.I., A. Inoue, R.Y., K.M., T.H., K.T., and H.S. are employees and/or stockholders of JCR Pharmaceuticals Co., Ltd."

Evidence found in paper:

"We thank Shin Nippon Biomedical Laboratories for conducting GLP-compliant toxicity studies using monkeys and Sapporo General Pathology Laboratory for histopathological analysis. We also thank all members of the Pharmacology & Toxicology Unit, Research Division of 10.13039/100019522JCR Pharmaceuticals for their support in this study. This research received no external funding."

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Last Updated: Aug 05, 2025