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Target intelligence / Profile preview
ATP-binding cassette sub-family A member 4 (ABCA4)
- Target
- ABCA4
- Molecular classification
- ABC transporter superfamily, ABC1 subfamily, Transporter
01
Overview
ATP-binding cassette sub-family A member 4 (ABCA4) is a retina-specific ABC transporter responsible for transporting N-retinylidene-phosphatidylethanolamine (N-retinylidene-PE), a toxic byproduct of the visual cycle, out of photoreceptor disc membranes. Mutations in ABCA4 are associated with several inherited retinal diseases, including Stargardt macular degeneration and cone-rod dystrophy, due to the accumulation of toxic retinoids.
- Other names
- ABCRCORD3ARMD2FFMRetinal-Specific Phospholipid-Transporting ATPase ABCA4Stargardt Disease Protein
02
Mechanism of action
Modulation of ABCA4 activity to enhance N-retinylidene-PE transport and reduce toxic retinoid accumulation is a potential therapeutic approach.
03
Biological functions
Transports N-retinylidene-phosphatidylethanolamineRetinoid flippaseClearance of all-trans-retinalClearance of excess 11-cis-retinal
04
Disease associations
Stargardt Macular DegenerationCone-Rod DystrophyRetinitis Pigmentosa
05
Safety considerations
Potential for off-target effects when developing therapies targeting ABCA4.Need for careful consideration of drug delivery to the retina.
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