TDP-43 in neurodegenerative disorders

Research output: Contribution to journalReview articlepeer-review

27 Scopus citations


Background: The number of neurodegenerative diseases associated with pathological aggregates of transactivation response element (TAR)-DNA-binding protein 43 (TDP-43) has increased, leading to the new designation 'TDP-43 proteinopathy.' Biochemically, TDP-43 proteinopathies are characterized by decreased solubility, hyperphosphorylation, and cleavage of TDP-43 into 25- and 35-kDa fragments, and by altered cellular localization. Objective: This review summarizes research characterizing the distribution of TDP-43 pathology in human postmortem brain tissue and discusses possible therapeutic strategies based on genetic and in vitro studies. Methods: We reviewed recent studies of TDP-43 proteinopathy. Results/conclusion: Given that several different mutations can lead to TDP-43 proteinopathies, including mutations in progranulin and valosin-containing protein, research is needed to decipher and potentially exploit the link between these mutations and TDP-43 pathology.

Original languageEnglish (US)
Pages (from-to)969-978
Number of pages10
JournalExpert Opinion on Biological Therapy
Issue number7
StatePublished - Jul 2008


  • Amyotropic lateral sclerosis
  • Frontotemporal dementia
  • Progranulin
  • TAR DNA binding protein-43 (TDP-43)
  • Valosin-containing protein

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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