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Divergent phenotypes in Gaucher disease implicate the role of modifiers

Background: Gaucher disease is classified into neuronopathic and non-neuronopathic forms with wide phenotypic variation among patients sharing the same genotype. While homozygosity for the common L444P allele usually correlates with the neuronopathic forms, how a defined genotype leads to a phenotyp...

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Autores principales: Goker-Alpan, O, Hruska, K, Orvisky, E, Kishnani, P, Stubblefield, B, Schiffmann, R, Sidransky, E
Formato: Artigo
Lenguaje:Inglês
Publicado: BMJ Group 2005
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC1736082/
https://ncbi.nlm.nih.gov/pubmed/15937077
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jmg.2004.028019
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