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
| Autor: | Laoharawee, Kanut, Podetz-Pedersen, Kelly M., Nguyen, Tam T., Singh, Sajya M., Smith, Miles C., Belur, Lalitha R., Low, Walter C., Kozarsky, Karen F., McIvor, R. Scott |
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| Zdroj: | In Molecular Genetics and Metabolism Reports March 2023 34 |
| Databáze: | ScienceDirect |
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