Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome.

Authors:
Laoharawee K; Podetz-Pedersen KM; Nguyen TT; Singh SM; Smith MC and 4 more

Journal:
Mol Genet Metab Rep

Publication Year: 2023

DOI:
10.1016/j.ymgmr.2023.100956

PMCID:
PMC9871739

PMID:
36704405

View on PubMed View on PMC View Publisher
Journal Information

Full Title: Mol Genet Metab Rep

Abbreviation: Mol Genet Metab Rep

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Genetics & Heredity

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

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Transparency Score
2/6
33.3% Transparent
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Evidence found in paper:

"The authors thank Dr. Joseph Muenzer for providing the IDS KO strain, and the University of Minnesota NINDS Behavioral Phenotyping Core. The work described in this manuscript was supported by 10.13039/100013394REGENXBIO."

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Last Updated: Aug 05, 2025