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Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy

Mucopolysaccharidosis type II (MPSII) is a pediatric lysosomal storage disease caused by deficiencies in the IDS (iduronate-2-sulfatase) gene resulting in accumulation of glycosaminoglycans, multisystem disease, and profound neurodegeneration in severe forms. Although enzyme replacement therapy is a...

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Detalles Bibliográficos
Autores principales:	Ellison, Stuart, Liao, Aiyin, Gleitz, Hélène F.E., Parker, Helen, Booth, Laura, Robinson, John, Wood, Shaun, Taylor, Jessica, Holley, Rebecca, Bigger, Brian W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10618237/ https://www.ncbi.nlm.nih.gov/pubmed/37920237 http://dx.doi.org/10.1016/j.omtm.2023.101127

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