TDP43 (TAR DNA-binding protein 43) is involved in transcriptional regulation and exon splicing (1,2). While normal TDP43 is a nuclear protein, pathological TDP43 is a component of insoluble aggregates in patients with frontotemporal lobar degeneration (FTLD) and amyotrophic lateral sclerosis (ALS). In these disorders, TDP43 is abnormally ubiquitinated, phosphorylated and cleaved to generate carboxy-terminal fragments that are sequestered as insoluble aggregates in neuronal nuclei, perikarya, and neurites (3,4). Additionally, TDP43 inhibits the expression of the HIV-1 gene and regulates CFTR gene splicing (1,5).
