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Potocki-Lupski syndrome

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Last updated 19d ago

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A child presents with hypotonia, failure to thrive, and developmental delay. CMA reveals a 3.7 Mb duplication at 17p11.2, the reciprocal of the Smith-Magenis deletion region.

AD (de novo); 17p11.2 duplication involving RAI1 (reciprocal of Smith-Magenis syndrome deletion). Most duplications are in tandem.

  • Infantile hypotonia and failure to thrive
  • Developmental delay and intellectual disability (mild to moderate)
  • Autism spectrum features (common)
  • Cardiovascular anomalies (structural heart defects in ~40%)
  • Sleep-disordered breathing / obstructive sleep apnea
  • Generally milder phenotype than Smith-Magenis
  • Chromosomal microarray (CMA): detects 17p11.2 duplication
  • Not detected by standard karyotype (submicroscopic)
  • Early intervention and therapies (speech, OT, PT)
  • Echocardiogram
  • Sleep study for sleep-disordered breathing
  • Growth monitoring

PLUS vs MINUS, the reciprocal pair at 17p11.2:

  • Potocki-Lupski = PLUS = duplication, three copies of the region
  • Smith-Magenis, said "sMIth-mageNUS" = MINUS = deletion, one copy

Same locus, opposite dose: the duplication and the deletion are reciprocal products of the same misalignment.

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