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transactive response (TAR) DNA-binding protein 43; TDP-43 (TARDBP TDP43)

Function: - DNA-binding protein & RNA-binding protein - regulates transcription & splicing - binds specifically to pyrimidine-rich motifs of TAR DNA & to single stranded TG repeated sequences - binds to RNA, specifically to UG repeated sequences with a minimun of 6 contiguous repeats - involved in the regulation of CFTR splicing - may also be involved in microRNA biogenesis, apoptosis & cell division. Structure: - contains 2 RRM domains (RNA recognition motif) Compartment: nucleus Expression: - ubiquitously expressed - expression is high in pancreas, placenta, lung, genital tract & spleen Pathology: 1) can repress HIV-1 transcription by binding to the HIV-1 long terminal repeat 2) role in the regulation of CFTR splicing - promotes CFTR exon 9 skipping by binding to the UG repeated motifs in the polymorphic region near the 3' splice site - this aberrant splicing is associated with pathological features typical of cystic fibrosis 3) hyperphosphorylated, ubiquitinated & cleaved to generate C-terminal fragments in hippocampus, neocortex & spinal cord of patients with amyotrophic lateral sclerosis (ALS) 4) absent from the nucleus of affected neurons in ALS but is the primary component of cytoplasmic ubiquitin-positive inclusion bodies 5) defects in TARDBP are the cause of ALS type 10 6) found in inclusions & neurites of patients with chronic traumatic encephalopathy 7) deposited in brain of patients with frontotemporal dementia (FTD) associated with motor neuron disease 8) associated with proteinopathy in over 20% of people > 80 years, limbic-predominant age-related TDP-43 encephalopathy (LATE) & limbic-predominant amnestic neurodegenerative syndrome (LANS) 9) a misfolded protein relatively common in association with other misfolded proteins amyloid-beta, PHF-tau, & alpha-synuclein in older adults [2] - the 4 misfolded proteins appear associated with cognitive impairment that typically progresses to severe dementia

Related

TARDBP gene mutation

General

DNA-binding protein phosphoprotein RNA-binding protein

Properties

SIZE: entity length = 414 aa MW = 45 kD COMPARTMENT: cytoplasm cell nucleus MOTIF: Ser phosphorylation site {S91} Ser phosphorylation site {S92} RNP motif NAME: RNP motif SITE: 104-200 FOR-BINDING-OF: ribonucleic acid MOTIF: ribonucleoprotein-1 motif NAME: ribonucleoprotein-1 motif FOR-BINDING-OF: ribonucleic acid MOTIF: ribonucleoprotein-1 motif ribonucleoprotein-2 motif ribonucleoprotein-2 motif FOR-BINDING-OF: ribonucleic acid MOTIF: ribonucleoprotein-1 motif ribonucleoprotein-2 motif RNP motif NAME: RNP motif SITE: 191-262 FOR-BINDING-OF: ribonucleic acid MOTIF: ribonucleoprotein-1 motif NAME: ribonucleoprotein-1 motif FOR-BINDING-OF: ribonucleic acid MOTIF: ribonucleoprotein-1 motif ribonucleoprotein-2 motif ribonucleoprotein-2 motif FOR-BINDING-OF: ribonucleic acid MOTIF: ribonucleoprotein-1 motif ribonucleoprotein-2 motif glycine-rich region {274-413}

Database Correlations

OMIM correlations MORBIDMAP 605078 UniProt Q13148 Pfam PF00076 Entrez Gene 23435 Kegg hsa:23435

References

  1. UniProt :accession Q13148
  2. Karanth S, Nelson PT, Katsumata Y et al Prevalence and Clinical Phenotype of Quadruple Misfolded Proteins in Older Adults. JAMA Neurol. Published online June 22, 2020 PMID: 32568358 Free PMC article https://jamanetwork.com/journals/jamaneurology/fullarticle/2767373