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Target intelligence / Profile preview
Cystinosin (CTNS)
- Target
- CTNS
- Molecular classification
- Transporter, Lysosomal membrane protein
01
Overview
Cystinosin is a lysosomal membrane protein encoded by the CTNS gene. It functions as an H+-driven symporter responsible for exporting cystine from lysosomes into the cytosol. This process is essential for cellular amino acid recycling and overall lysosomal homeostasis. Mutations in CTNS cause cystinosis, a lysosomal storage disease characterized by abnormal accumulation of cystine crystals within cells.
- Other names
- CTNS-LSBPQLC4
02
Mechanism of action
Cysteamine reacts with intralysosomal cystine, forming compounds that can exit lysosomes via alternative transporters.
03
Biological functions
Cystine transportAmino acid recyclingLysosomal homeostasisRegulation of melanin production (possible)
04
Disease associations
CystinosisLysosomal storage disease
05
Safety considerations
Nephrotoxicity (Cysteamine)Gastrointestinal side effects (Cysteamine)Neurological side effects (Cysteamine)Monitoring of cystine levels is essentialDisease progression despite treatmentVariability in treatment response
06
Interacting drugs
Cysteamine
07
Biomarkers
Intracellular cystine levelsKidney function markers (e.g., creatinine, GFR)Growth parametersThyroid hormone levels
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