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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...
में बचाया:
| मुख्य लेखकों: | , , , |
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| स्वरूप: | Artigo |
| भाषा: | Inglês |
| प्रकाशित: |
National Academy of Sciences
2008
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| विषय: | |
| ऑनलाइन पहुंच: | 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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