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In vivo correction of a Menkes disease model using antisense oligonucleotides
Although the molecular basis of many inherited metabolic diseases has been defined, the availability of effective therapies in such disorders remains problematic. Menkes disease is a fatal neurodegenerative disorder due to loss-of-function mutations in the ATP7A gene encoding a copper-transporting P...
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| Main Authors: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
National Academy of Sciences
2008
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2268804/ https://ncbi.nlm.nih.gov/pubmed/18316734 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0710865105 |
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