{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,14]],"date-time":"2025-05-14T00:57:52Z","timestamp":1747184272820,"version":"3.40.5"},"reference-count":97,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,1,19]],"date-time":"2024-01-19T00:00:00Z","timestamp":1705622400000},"content-version":"vor","delay-in-days":18,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100008731","name":"Association Europ\u00e9enne contre les Leucodystrophies","doi-asserted-by":"publisher","award":["ELA 2020\u2010004I3"],"award-info":[{"award-number":["ELA 2020\u2010004I3"]}],"id":[{"id":"10.13039\/501100008731","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Dev Growth Differ"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Inherited leukodystrophies are genetic disorders characterized by abnormal white matter in the central nervous system. Although individually rare, there are more than 400 distinct types of leukodystrophies with a cumulative incidence of 1 in 4500 live births. The pathophysiology of most leukodystrophies is poorly understood, there are treatments for only a few, and there is significant morbidity and mortality, suggesting a critical need for improvements in this field. A variety of animal, cell, and induced pluripotent stem cell\u2010derived models have been developed for leukodystrophies, but with significant limitations in all models. Many leukodystrophies lack animal models, and extant models often show no or mixed recapitulation of key phenotypes. Zebrafish (<jats:italic>Danio rerio<\/jats:italic>) have become increasingly used as disease models for studying leukodystrophies due to their early onset of disease phenotypes and conservation of molecular and neurobiological mechanisms. Here, we focus on reviewing new zebrafish disease models for leukodystrophy or models with recent progress. This includes discussion of leukodystrophy with vanishing white matter disease, X\u2010linked adrenoleukodystrophy, Zellweger spectrum disorders and peroxisomal disorders, PSAP deficiency, metachromatic leukodystrophy, Krabbe disease, hypomyelinating leukodystrophy\u20108\/4H leukodystrophy, Aicardi\u2013Gouti\u00e8res syndrome, RNASET2\u2010deficient cystic leukoencephalopathy, hereditary diffuse leukoencephalopathy with spheroids\u20101 (CSF1R\u2010related leukoencephalopathy), and ultra\u2010rare leukodystrophies. Zebrafish models offer important potentials for the leukodystrophy field, including testing of new variants in known genes; establishing causation of newly discovered genes; and early lead compound identification for therapies. There are also unrealized opportunities to use humanized zebrafish models which have been sparsely explored.<\/jats:p>","DOI":"10.1111\/dgd.12907","type":"journal-article","created":{"date-parts":[[2024,1,19]],"date-time":"2024-01-19T08:18:30Z","timestamp":1705652310000},"page":"21-34","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Progress in leukodystrophies with zebrafish"],"prefix":"10.1111","volume":"66","author":[{"given":"Hung\u2010Yu","family":"Shih","sequence":"first","affiliation":[{"name":"Department of Pediatrics University of Utah School of Medicine  Salt Lake City Utah USA"},{"name":"Department of Biological Sciences Utah Tech University  Saint George Utah USA"},{"name":"Center for Precision &amp; Functional Genomics, Utah Tech University  Saint George Utah USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Quentin","family":"Raas","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Utah School of Medicine  Salt Lake City Utah USA"},{"name":"Laboratory of Translational Research for Neurological Disorders Imagine Institute, Universit\u00e9 de Paris, INSERM UMR 1163  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8775-147X","authenticated-orcid":false,"given":"Joshua L.","family":"Bonkowsky","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Utah School of Medicine  Salt Lake City Utah USA"},{"name":"Center for Personalized Medicine, Primary Children's Hospital  Salt Lake City Utah USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,1,19]]},"reference":[{"key":"e_1_2_14_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.50826"},{"key":"e_1_2_14_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.brainres.2015.10.011"},{"key":"e_1_2_14_4_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073817751804"},{"key":"e_1_2_14_5_1","doi-asserted-by":"publisher","DOI":"10.1042\/BST0330433"},{"key":"e_1_2_14_6_1","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.048925"},{"key":"e_1_2_14_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-022-02440-5"},{"key":"e_1_2_14_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/glia.23598"},{"key":"e_1_2_14_9_1","unstructured":"Bernard G. &Vanderver A.(2012).POLR3\u2010Related Leukodystrophy. Aug 2 [updated 2017 May 11]. In: Adam MP Feldman J Mirzaa GM Pagon RA Wallace SE Bean LJH Gripp KW Amemiya A editors. GeneReviews\u00ae [Internet]. Seattle (WA): University of Washington Seattle; 1993\u20132023."},{"key":"e_1_2_14_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.coviro.2016.08.001"},{"key":"e_1_2_14_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-023-04700-3"},{"key":"e_1_2_14_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1399-0004.2008.01058.x"},{"key":"e_1_2_14_13_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2021-053126"},{"key":"e_1_2_14_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41593-020-0703-x"},{"key":"e_1_2_14_15_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13041-019-0479-7"},{"key":"e_1_2_14_16_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13041-017-0294-y"},{"key":"e_1_2_14_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2020.02.021"},{"key":"e_1_2_14_18_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.aat5314"},{"key":"e_1_2_14_19_1","doi-asserted-by":"crossref","unstructured":"Coulombe B. Derksen A. La Piana R. Brais B. 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