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N Engl J Med. 2010 Aug 26;363(9):852-63. doi: 10.1056/NEJMra0907180.

New therapeutic approaches to mendelian disorders.

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  • 1McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, 733 N. Broadway St., Baltimore, MD 21205, USA. hdietz@jhmi.edu
PMID:
20818846
[PubMed - indexed for MEDLINE]
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