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Alagille syndrome: Genetics and Functional Models

PURPOSE OF REVIEW: We review the genetics of the autosomal dominant, multi-system disorder, Alagille syndrome and provide a summary on how current functional models and emerging biotechnologies are equipped to guide scientists towards novel therapies. The importance of haploinsufficiency as a diseas...

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Detalhes bibliográficos
Publicado no:Curr Pathobiol Rep
Main Authors: Gilbert, Melissa A., Spinner, Nancy B.
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5736143/
https://ncbi.nlm.nih.gov/pubmed/29270332
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40139-017-0144-8
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