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Tetratricopeptide repeat domain 21B (TTC21B)
- Target
- TTC21B
- Molecular classification
- Other (specifically, ciliary transport protein, component of IFT-A complex)
01
Overview
Tetratricopeptide repeat domain 21B (TTC21B) is a protein-coding gene encoding a component of the intraflagellar transport complex A (IFT-A) required for retrograde ciliary protein trafficking, including the movement of G protein-coupled receptors (GPCRs) within cilia. TTC21B is characterized by several tetratricopeptide repeat (TPR) domains, is localized to the ciliary axoneme, and plays crucial roles in ciliary function and maintenance. Mutations in TTC21B disrupt ciliary transport and are causally linked to a spectrum of ciliopathies including nephronophthisis 12 (NPHP12), Jeune asphyxiating thoracic dystrophy (JATD/SRTD4), and certain forms of retinal degeneration; it can also act as a genetic modifier in multi-gene ciliopathy syndromes. There are no therapeutics directly targeting TTC21B at this time, but clinical assessment of its variants is used for diagnosis and genetic counseling in relevant inherited disorders.
- Other names
- TTC21BATD4IFT139JBTS11NPHP12SRTD4THM1Nbla10696IFT139BFAP60FLA17Tetratricopeptide repeat protein 21B
02
Biological functions
Retrograde intraflagellar transport in ciliaRegulation of ciliary protein localization and traffickingNegative modulation of Sonic hedgehog (SHH) signal transduction
03
Disease associations
Ciliopathies (collective group, including genetic and functional contributors)Nephronophthisis 12 (NPHP12)Jeune asphyxiating thoracic dystrophy (JATD; short-rib thoracic dysplasia 4)Retinal degeneration
04
Safety considerations
Mutations can cause or modify severe and often multi-organ genetic disorders (ciliopathies), including renal, skeletal, and retinal pathologies
05
Biomarkers
TTC21B mutations as biomarkers for certain ciliopathies (e.g., NPHP12, JATD)
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