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Target intelligence / Profile preview
ATP Binding Cassette Subfamily A Member 4 (ABCA4)
- Target
- ABCA4
- Molecular classification
- Transporter, ATP-binding cassette transporter, ABC1 subfamily
01
Overview
ATP binding cassette subfamily A member 4 (ABCA4) is a retina-specific membrane protein and an ABC transporter that plays a crucial role in the visual cycle. It transports N-retinylidene-phosphatidylethanolamine across photoreceptor disc membranes, preventing the accumulation of toxic retinoids. Mutations in ABCA4 are associated with several retinal diseases, including Stargardt disease, cone-rod dystrophy and retinitis pigmentosa.
- Other names
- ABCRRIM protein / RIM ABC transporterRetina-specific ABC transporterPhotoreceptor rim proteinStargardt disease protein
02
Mechanism of action
Inhibiting ABCA4 function leads to accumulation of toxic retinoids, while enhancing ABCA4 function promotes clearance of retinoids.
03
Biological functions
Transporter activityATPase-coupled transmembrane transporter activityRetinoid transportVisual cycle
04
Disease associations
Stargardt disease type ICone-Rod DystrophyRetinitis PigmentosaAge-related Macular Degeneration Type II
05
Safety considerations
Potential for retinal toxicity due to altered retinoid metabolismSeverity of disease phenotype dependent on specific ABCA4 mutation
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