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    • A generalizable pre-clinical research approach for orphan disease therapy 

      Beaulieu, Chandree L; Samuels, Mark E; Ekins, Sean; McMaster, Christopher R; Edwards, Aled M; Krainer, Adrian R; Hicks, Geoffrey G; Frey, Brendan J; Boycott, Kym M; MacKenzie, Alex E (2012-06-15)
      AbstractWith the advent of next-generation DNA sequencing, the pace of inherited orphan disease gene identification has increased dramatically, a situation that will continue for at least the next several years. At present, ...