AIPL1-related retinopathy

MONDO:0100438

A retinopathy caused by biallelic variants in the AIPL1 gene.

Also known as: AIPL1 retinopathy, AIPL1 Leber congenital amaurosis, LCA4, Leber congenital amaurosis 4, Leber congenital amaurosis caused by mutation in AIPL1, Leber congenital amaurosis type 4, amaurosis congenita of Leber, type 4, cone-rod dystrophy

26 clinical trials for this condition and its sub-types, 1 tagged with AIPL1-related retinopathy itself.

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Where it sits in the disease tree

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↑ Inherited retinal dystrophy (510)

Sub-types of AIPL1-related retinopathy

Leber congenital amaurosis 4 0 trials

Including sub-types (26) Tagged with AIPL1-related retinopathy (1)

Trials to join now! 1

Gene therapy hope for kids and adults with rare Blindness-Causing mutation

Disease control Recruiting now

This early-phase study tests a gene therapy called LX107 for people with a rare inherited eye disease caused by mutations in the AIPL1 gene. The treatment is given as an injection under the retina to try to improve vision and slow disease progression. The study will include 13 ad…

Early phase 1 • Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine • Aim: Disease control

Last updated Jun 27, 2026 11:00 UTC

AIPL1-related retinopathy

Where it sits in the disease tree

Gene therapy hope for kids and adults with rare Blindness-Causing mutation