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Delivering gene therapy for mucopolysaccharide diseases

Mucopolysaccharide diseases are a group of paediatric inherited lysosomal storage diseases that are caused by enzyme deficiencies, leading to a build-up of glycosaminoglycans (GAGs) throughout the body. Patients have severely shortened lifespans with a wide range of symptoms including inflammation,...

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Detalles Bibliográficos
Autores principales:	Wood, Shaun R., Bigger, Brian W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2022
Materias:	Molecular Biosciences
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9511407/ https://www.ncbi.nlm.nih.gov/pubmed/36172050 http://dx.doi.org/10.3389/fmolb.2022.965089

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