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Alleles with more than one mutation can complicate genotype/phenotype studies in Mendelian disorders: Lessons from Gaucher disease

Autosomal resessive Mendelian disorders usually result from two inherited disease-causing mutations. However, this is not always the case. Focusing on Gaucher disease, which results from mutations in GBA1, we found that more comprehensive genotyping revealed important exceptions. For example, patien...

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Dettagli Bibliografici
Pubblicato in:Mol Genet Metab
Autori principali: Hassan, Shahzeb, Lopez, Grisel, Stubblefield, Barbara K., Tayebi, Nahid, Sidransky, Ellen
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2018
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6178817/
https://ncbi.nlm.nih.gov/pubmed/29980418
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2018.06.013
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