Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.

doi:10.1172/jci.insight.145445

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Article

Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.

JCI insight - 8 Oct 2021

Arguello Annie, Meisner René, Thomsen Elliot R, Nguyen Hoang N, Ravi Ritesh, Simms Jeffrey, Lo Iris, Speckart Jessica, Holtzman Julia, Gill Thomas M, Chan Darren, Cheng Yuhsiang, Chiu Chi-Lu, Dugas Jason C, Fang Meng, Lopez Isabel A, Solanoy Hilda, Tsogtbaatar Buyankhishig, Zhu Yuda, Bhalla Akhil, Henne Kirk R, Henry Anastasia G, Delucchi Anthony, Costanzo Simona, Harris Jeffrey M, Diaz Dolores, Scearce-Levie Kimberly, Sanchez Pascal E

Abstract excerpt

Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder caused by deficiency of the iduronate-2-sulfatase (IDS) enzyme, resulting in cellular accumulation of glycosaminoglycans (GAGs) throughout the body. Treatment of MPS II remains a considerable challenge as current enzyme replacement therapies do not adequately control many aspects of the disease, including skeletal and neurological...

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PMID: 34622797
DOI: 10.1172/jci.insight.145445
PMCID: PMC8525587

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