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Target intelligence / Profile preview
ATP-binding cassette sub-family D member 1 (ABCD1)
- Target
- ABCD1
- Molecular classification
- ATP-binding cassette transporters, ABC transporter, Half-transporter
01
Overview
ATP-binding cassette sub-family D member 1 (ABCD1) encodes adrenoleukodystrophy protein (ALDP), a peroxisomal membrane protein. ALDP is crucial for importing very long-chain fatty acids (VLCFAs) into peroxisomes for degradation. Mutations in ABCD1 cause X-linked adrenoleukodystrophy (X-ALD), a severe neurodegenerative disorder characterized by progressive cognitive decline, motor dysfunctions, and hormonal imbalances due to adrenal insufficiency. ABCD1 functions as a half-transporter, requiring dimerization for activity.
- Other names
- ALDPALDAMNATP-binding cassette sub-family D (ALD), member 1ABCD1_HUMAN
02
Biological functions
Transports VLCFAs into peroxisomesFatty acid catabolismRegulation of microsomal fatty acid elongation
03
Disease associations
X-linked AdrenoleukodystrophyNeurodegenerative diseaseAdrenal insufficiency
04
Safety considerations
Variability in disease severity and progression despite identical mutationsPotential for inflammatory responses and demyelination in the central nervous system
05
Biomarkers
VLCFA levelsALDP protein levelsGenetic mutations in ABCD1
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