Rapid Resolution of Blended or Composite Multigenic Disease in Infants by Whole-Exome Sequencing.

Theunissen, Tom E J; Sallevelt, Suzanne C E H; Hellebrekers, Debby M E I; de Koning, Bart; Hendrickx, Alexandra T M; van den Bosch, Bianca J C; Kamps, Rick; Schoonderwoerd, Kees et al. · J Pediatr · 2017

case_report · Level V

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Abstract

Whole-exome sequencing identified multiple genetic causes in 2 infants with heterogeneous disease. Three gene defects in the first patient explained all symptoms, but manifestations were overlapping (blended phenotype). Two gene defects in the second patient explained nonoverlapping symptoms (composite phenotype). Whole-exome sequencing rapidly and comprehensively resolves heterogeneous genetic disease.

Medical subject headings