ABCA4 (P78363) variants and mutations

ABCA4 (also known as P78363) is a human protein-coding gene encoding a retinal-specific phospholipid-transporting ATPase protein. It flips retinal-derived lipid adducts across photoreceptor disc membranes so they can be cleared during the visual cycle. Biallelic loss-of-function variants cause Stargardt disease and can also produce cone-rod dystrophy or retinitis pigmentosa. This analysis covers 3,721 ABCA4 variants and mutations. Of these, 91% have computational variant effect predictions. Disease context includes severe early-childhood-onset retinal dystrophy, Stargardt disease, and cone-rod dystrophy 3. Example ABCA4 variants include M1I, M1L, and M1T.

Variant analysis overview

Variant and mutation evidence

Clinical, disease, and population context

Protein structure and variant hotspots

Data sources

Evidence in this analysis draws on EBI Proteins Variation, UniProt, gnomAD v4, EuropePMC, 3D Hotspot Analysis, Interaction Network Analysis, Protein Data Bank, AlphaFold DB, gnomAD constraint, Open Targets, ClinGen, MaveDB, LitVar.

Notable ABCA4 variants

Examples include M1I, M1L, M1T, M1V, G2D, G2R, F3S, V4A. Listed records include available protein-change notation, database identifiers, clinical classifications, computational predictions, population evidence, experimental measurements, and disease context.