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Readthrough Strategies for Therapeutic Suppression of Nonsense Mutations in Inherited Metabolic Disease
Inherited metabolic diseases (IMDs) belong to the group of rare diseases due to their low individual prevalence. Most of them are inherited in autosomal recessive fashion and represent good candidates for novel therapeutical strategies aimed at recovering partial enzyme function as they lack an effe...
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| Main Authors: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
S. Karger AG
2012
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3531923/ https://ncbi.nlm.nih.gov/pubmed/23293581 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1159/000343086 |
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