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• W1989959309 startingPage "852" @default.
• W1989959309 abstract "Thanks to the power of a method to identify etiologic mutations (and hence “causative” genes) in mendelian disease, the molecular mechanisms that give rise to many such diseases are now known. This knowledge has fueled new therapeutic approaches, which are reviewed in this article, the third in the Genomic Medicine series." @default.
• W1989959309 created "2016-06-24" @default.
• W1989959309 creator A5038027788 @default.
• W1989959309 date "2010-08-26" @default.
• W1989959309 modified "2023-10-17" @default.
• W1989959309 title "New Therapeutic Approaches to Mendelian Disorders" @default.
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• W1989959309 doi "https://doi.org/10.1056/nejmra0907180" @default.
• W1989959309 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/20818846" @default.
• W1989959309 hasPublicationYear "2010" @default.
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