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The cytoplasmic aggregation of TDP-43 (TAR DNA-binding protein 43), an RNA-binding protein, is considered a hallmark of ALS pathology, found in nearly all postmortem cases of ALS. TDP-43 is normally primarily nuclear, where it has a widespread role in gene regulation. Mutations, extrinsic stressors, and alterations in RNA homeostasis in ALS lead to nuclear depletion of TDP-43 and the formation of cytosolic TDP-43 aggregates. This causes multiple downstream effects on neuronal function and degeneration as well as gene expression. TDP-43 is a promising target as a biomarker, as it is found to be elevated in the biofluids of ALS patients, and its cytoplasmic aggregation can also be observed in peripheral tissues; however, methodological variability and technical limitations currently preclude the establishment of TDP-43 as a standalone biomarker. There are also promising therapeutic strategies in development targeting TDP-43 pathology, but a critical challenge that remains is achieving a balance between eliminating toxic aggregates and preserving the essential functions of TDP-43. In summary, with further research, considering TDP-43 pathology in ALS gives hope for finding future novel diagnostics and therapeutics for ALS.<\/jats:p>","DOI":"10.1042\/bst20260896","type":"journal-article","created":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T08:06:20Z","timestamp":1782893180000},"page":"901-913","update-policy":"https:\/\/doi.org\/10.1042\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["TDP-43 proteinopathy as a biomarker and therapeutic target in amyotrophic lateral sclerosis"],"prefix":"10.1042","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9352-4908","authenticated-orcid":false,"given":"Eleni","family":"Christoforidou","sequence":"first","affiliation":[{"name":"Department of Neuroscience, School of Life Sciences, University of Sussex, Brighton, U.K."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily","family":"McFagan","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, School of Life Sciences, University of Sussex, Brighton, U.K."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6142-3811","authenticated-orcid":false,"given":"Martha","family":"McLaughlin","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, School of Life Sciences, University of Sussex, Brighton, U.K."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5262-7150","authenticated-orcid":false,"given":"Majid","family":"Hafezparast","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, School of Life Sciences, University of Sussex, Brighton, U.K."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"288","published-online":{"date-parts":[[2026,7,1]]},"reference":[{"key":"2026070104053002000_B1","doi-asserted-by":"publisher","first-page":"118","DOI":"10.1159\/000351153","article-title":"Global epidemiology of amyotrophic lateral sclerosis: a systematic review of the published literature","volume":"41","author":"Chio","year":"2013","journal-title":"Neuroepidemiology"},{"key":"2026070104053002000_B2","doi-asserted-by":"publisher","first-page":"942","DOI":"10.1016\/S0140-6736(10)61156-7","article-title":"Amyotrophic lateral sclerosis","volume":"377","author":"Kiernan","year":"2011","journal-title":"Lancet"},{"key":"2026070104053002000_B3","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1038\/nn.3584","article-title":"State of play in amyotrophic lateral sclerosis genetics","volume":"17","author":"Renton","year":"2014","journal-title":"Nat. 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