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Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive and severe debilitating neurological dysfunct...

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Detalles Bibliográficos
Autores principales:	Rouse, Courtney J., Jensen, Victoria N., Heldermon, Coy D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wolters Kluwer - Medknow 2023
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10503619/ https://www.ncbi.nlm.nih.gov/pubmed/37488890 http://dx.doi.org/10.4103/1673-5374.377606

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