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Target intelligence / Profile preview
Transthyretin (TTR)
- Target
- TTR
- Molecular classification
- Transport protein, Carrier protein
01
Overview
Transthyretin (TTR) is a homotetrameric serum and cerebrospinal fluid protein that transports thyroid hormone (T4) and retinol. It is primarily synthesized in the liver and choroid plexus. Mutations or misfolding of TTR are associated with amyloidosis, particularly hereditary ATTRv and wild-type ATTRwt, leading to organ damage primarily affecting the nerves and heart. TTR is considered a therapeutic target for stabilizing drugs like Tafamidis.
- Other names
- PrealbuminThyroxine-binding prealbumin
02
Mechanism of action
Tafamidis stabilizes the transthyretin tetramer, preventing dissociation and misfolding, thus inhibiting amyloid fibril formation.
03
Biological functions
Thyroid hormone transportRetinol transportNeuroprotectionAmyloid-beta interactionDetoxification
04
Disease associations
Hereditary transthyretin amyloidosis (ATTRv)Wild-type ATTR amyloidosis (ATTRwt)AmyloidosisNeurodegenerative diseaseCardiomyopathyPolyneuropathy
05
Safety considerations
Drug interactionsPotential for off-target effectsMonitoring for disease progressionAdverse events associated with TTR-stabilizing drugs
06
Interacting drugs
Tafamidis
1 more in the full profile.
07
Biomarkers
TTR levels in serumTTR mutationsCardiac biomarkers (for ATTRwt)Neuropathy assessment (for ATTRv)
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