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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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Detalhes bibliográficos
Publicado no:Mamm Genome
Main Authors: Vashi, Neeti, Justice, Monica J.
Formato: Artigo
Idioma:Inglês
Publicado em: Springer US 2019
Assuntos:
Acesso em linha: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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