LRAT
Lecithin retinol acyltransferase; essential for the visual cycle (same pathway as RPE65)
Active Clinical Trials
LRAT-LCA shares the visual-cycle defect of RPE65, so it was targeted with an oral synthetic retinoid (QLT091001 / zuretinol acetate) rather than gene therapy. A Phase 1b trial in RPE65- or LRAT-deficient LCA/RP reported transient improvements in visual field and acuity (Koenekoop et al., Lancet 2014). A later Phase 2/3 trial was withdrawn and no therapy is approved. AAV LRAT gene therapy has not entered clinical trials.
Sourced & cited — not yet expert-reviewed
Every statement here is backed by a cited primary source, but a specialist has not formally reviewed this page yet.
4q32.1
Autosomal Recessive
<1% of LCA cases
Key Clinical Features
- 1Visual cycle defect (similar pathway to RPE65)
- 2Severe early visual loss
- 3Potential gene therapy target
Clinical Trials
Oral QLT091001 (zuretinol) in RPE65/LRAT-deficient LCA/RP
QLT Inc. · Phase 1b
Zuretinol Acetate for RPE65/LRAT-deficient IRD
Retinagenix Holdings · Phase 2/3