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

Hunter syndrome is a rare x-linked recessive genetic disorder that affects lysosomal metabolism due to deficiency of iduronate-2-sulfatase (IDS), with subsequent accumulation of glycosaminoglycans heparan and dermatan sulfates (GAG). Enzyme replacement therapy is the only FDA-approved remedy and is...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9871739/
https://www.ncbi.nlm.nih.gov/pubmed/36704405
http://dx.doi.org/10.1016/j.ymgmr.2023.100956

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