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DTHD1

DTHD1

Death domain-containing protein 1; exact role in vision is unknown. Identified by Abu-Safieh et al. (2013) through autozygome analysis and exome sequencing in a consanguineous family with LCA and mild muscular dystrophy.

Henüz Hedefli Tedavi Yok

Kromozomal Lokus

4p14

Kalıtım

Otozomal Resesif

Sıklık

<1% LCA vakalarının

Temel Klinik Özellikler

  • 1No official LCA subtype number assigned
  • 2Protein function in retina not yet characterized
  • 3Identified in a single family with LCA and mild muscular dystrophy
  • 4c.2T-C mutation causes >4-fold reduction in protein abundance

Sendromik İlişkiler

DTHD1 mutasyonlu hastalar diğer organların olası tutulumu açısından izlenmelidir:

  • Mild to moderate muscular dystrophy (in the original family)