{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T10:38:49Z","timestamp":1753871929873,"version":"3.41.2"},"reference-count":34,"publisher":"Portland Press Ltd.","issue":"2","license":[{"start":{"date-parts":[[2019,9,9]],"date-time":"2019-09-09T00:00:00Z","timestamp":1567987200000},"content-version":"vor","delay-in-days":154,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["portlandpress.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2019,6,28]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>CLN6-Batten disease is a rare neurodegenerative disorder with no cure, characterized by accumulation of lipofuscin in the lysosome, glial activation, and neuronal death. Here we test the therapeutic efficacy of modulating collapsin response mediator protein 2 (CRMP2) activity via S-N-benzy-2-acetamido-3-methoxypropionamide ((S)-Lacosamide) in a mouse model of CLN6-Batten disease. Promisingly, mouse neuronal cultures as well as Cln6 patient fibroblasts treated with varying concentrations of (S)-Lacosamide showed positive restoration of lysosomal associated deficits. However, while acute in vivo treatment enhanced glial activation in 3-month-old Cln6 mutant mice, chronic treatment over several months did not improve behavioral or long-term survival outcomes. Therefore, modulation of CRMP2 activity via (S)-Lacosamide alone is unlikely to be a viable therapeutic target for CLN6-Batten disease.<\/jats:p>","DOI":"10.1042\/ns20190001","type":"journal-article","created":{"date-parts":[[2019,3,29]],"date-time":"2019-03-29T15:55:15Z","timestamp":1553874915000},"update-policy":"https:\/\/doi.org\/10.1042\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Modulation of CRMP2 via (<i>S<\/i>)-Lacosamide shows therapeutic promise but is ultimately ineffective in a mouse model of CLN6-Batten disease"],"prefix":"10.1042","volume":"3","author":[{"given":"Katherine\u00a0A.","family":"White","sequence":"first","affiliation":[{"name":"Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, U.S.A."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jacob\u00a0T.","family":"Cain","sequence":"additional","affiliation":[{"name":"Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, U.S.A."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Helen","family":"Magee","sequence":"additional","affiliation":[{"name":"Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, U.S.A."},{"name":"Basic Biomedical Sciences, University of South Dakota Sanford School of Medicine, Vermillion, SD, U.S.A."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Seul\u00a0Ki","family":"Yeon","sequence":"additional","affiliation":[{"name":"Convergence Research Center for Dementia, Brain Science Institute, Korea Institute of Science and Technology, Seoul, South Korea"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ki\u00a0Duk","family":"Park","sequence":"additional","affiliation":[{"name":"Convergence Research Center for Dementia, Brain Science Institute, Korea Institute of Science and Technology, Seoul, South Korea"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rajesh","family":"Khanna","sequence":"additional","affiliation":[{"name":"Department of Pharmacology, University of Arizona, Tucson, AZ, U.S.A."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2504-4942","authenticated-orcid":false,"given":"Jill\u00a0M.","family":"Weimer","sequence":"additional","affiliation":[{"name":"Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, U.S.A."},{"name":"Department of Pediatrics, University of South Dakota, Sioux Falls, SD, U.S.A."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"288","published-online":{"date-parts":[[2019,4,8]]},"reference":[{"key":"2021111608023758000_B1","doi-asserted-by":"publisher","first-page":"2237","DOI":"10.1016\/j.bbadis.2015.05.011","article-title":"Genetics of the neuronal ceroid lipofuscinoses (Batten disease)","volume":"1852","author":"Mole","year":"2015","journal-title":"Biochim. 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