Target intelligence / Profile preview

TAR DNA-binding protein 43 (TDP-43)

Target
TDP-43
Molecular classification
DNA-binding protein, RNA-binding protein, Transcription factor
01

Overview

TAR DNA-binding protein 43 (TDP-43) is a highly conserved, predominantly nuclear protein that plays critical roles in gene expression regulation, RNA processing, and cellular stress responses. It is best known for its involvement in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD), where it forms pathological cytoplasmic inclusions.

Other names
transactive response DNA binding protein 43 kDaTARDBP
02

Biological functions

Transcription repressionAlternative splicing regulationmRNA stabilitymRNA transportMicroRNA processingDNA repairStress response
03

Disease associations

Amyotrophic Lateral Sclerosis (ALS)Frontotemporal Lobar Degeneration (FTLD)Neurodegenerative disease
04

Safety considerations

Aggregation propensityMislocalizationDisruption of RNA metabolism

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