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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...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
MDPI
2021
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| 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 |