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Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art

The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nerv...

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Detalles Bibliográficos
Autores principales: de Castro, María José, del Toro, Mireia, Giugliani, Roberto, Couce, María Luz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8430935/
https://www.ncbi.nlm.nih.gov/pubmed/34502108
http://dx.doi.org/10.3390/ijms22179200