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Treating Rett syndrome: from mouse models to human therapies
Rare diseases are very difficult to study mechanistically and to develop therapies for because of the scarcity of patients. Here, the rare neuro-metabolic disorder Rett syndrome (RTT) is discussed as a prototype for precision medicine, demonstrating how mouse models have led to an understanding of t...
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| Udgivet i: | Mamm Genome |
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| Main Authors: | , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Springer US
2019
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6606665/ https://ncbi.nlm.nih.gov/pubmed/30820643 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00335-019-09793-5 |
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