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WhatIsLCA
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LCA13

RDH12

Retinol dehydrogenase; part of the visual cycle, converts all-trans-retinal

Preklinik Araştırma

Opus Genetics OPGx-RDH12 in partnership with Global RDH12 Alliance. IND filing planned late 2025, first patient expected Q4 2026.

Kromozomal Lokus

14q24.1

Kalıtım

Otozomal Resesif

Sıklık

~10% LCA vakalarının

OMIM #612712

Temel Klinik Özellikler

  • 1LCA phenotype with significant disease progression in adolescence
  • 2Early, peripherally progressive macular atrophy with watercolor-like pattern