{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T16:31:31Z","timestamp":1792081891827,"version":"4.4.0"},"reference-count":60,"publisher":"Public Library of Science (PLoS)","issue":"12","license":[{"start":{"date-parts":[[2024,12,5]],"date-time":"2024-12-05T00:00:00Z","timestamp":1733356800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["5R01NS111715-05"],"award-info":[{"award-number":["5R01NS111715-05"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["5R01NS111715-04"],"award-info":[{"award-number":["5R01NS111715-04"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014177","name":"Legacy of Angels Foundation","doi-asserted-by":"publisher","award":["1171367-1-92757"],"award-info":[{"award-number":["1171367-1-92757"]}],"id":[{"id":"10.13039\/100014177","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014177","name":"Legacy of Angels Foundation","doi-asserted-by":"publisher","award":["1171367-1-92757"],"award-info":[{"award-number":["1171367-1-92757"]}],"id":[{"id":"10.13039\/100014177","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:p>Krabbe disease (Kd) is a lysosomal storage disorder (LSD) caused by the deficiency of the lysosomal galactosylceramidase (GALC) which cleaves the myelin enriched lipid galactosylceramide (GalCer). Accumulated GalCer is catabolized into the cytotoxic lipid psychosine that causes myelinating cells death and demyelination which recruits microglia\/macrophages that fail to digest myelin debris and become globoid cells. Here, to understand the pathological mechanisms of Kd, we used induced pluripotent stem cells (iPSCs) from Kd patients to produce myelinating organoids and microglia. We show that Kd organoids have no obvious defects in neurogenesis, astrogenesis, and oligodendrogenesis but manifest early myelination defects. Specifically, Kd organoids showed shorter but a similar number of myelin internodes than Controls at the peak of myelination and a reduced number and shorter internodes at a later time point. Interestingly, myelin is affected in the absence of autophagy and mTOR pathway dysregulation, suggesting lack of lysosomal dysfunction which makes this organoid model a very valuable tool to study the early events that drive demyelination in Kd. Kd iPSC-derived microglia show a marginal rate of globoid cell formation under normal culture conditions that is drastically increased upon GalCer feeding. Under normal culture conditions, Kd microglia show a minor LAMP1 content decrease and a slight increase in the autophagy protein LC3B. Upon GalCer feeding, Kd cells show accumulation of autophagy proteins and strong LAMP1 reduction that at a later time point are reverted showing the compensatory capabilities of globoid cells. Altogether, this supports the value of our cultures as tools to study the mechanisms that drive globoid cell formation and the compensatory mechanism in play to overcome GalCer accumulation in Kd.<\/jats:p>","DOI":"10.1371\/journal.pone.0314858","type":"journal-article","created":{"date-parts":[[2024,12,5]],"date-time":"2024-12-05T13:56:19Z","timestamp":1733406979000},"page":"e0314858","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":13,"title":["Human iPSC-derived myelinating organoids and globoid cells to study Krabbe disease"],"prefix":"10.1371","volume":"19","author":[{"given":"Lisa Marie P.","family":"Evans","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8088-1298","authenticated-orcid":true,"given":"Joseph","family":"Gawron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fraser J.","family":"Sim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Laura","family":"Feltri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1042-1872","authenticated-orcid":true,"given":"Leandro N.","family":"Marziali","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2024,12,5]]},"reference":[{"issue":"11","key":"pone.0314858.ref001","doi-asserted-by":"crossref","first-page":"2835","DOI":"10.1002\/ajmg.a.35624","article-title":"Death rates in the U.S. due to Krabbe disease and related leukodystrophy and lysosomal storage diseases","volume":"158A","author":"AL Barczykowski","year":"2012","journal-title":"Am J Med Genet A"},{"issue":"1","key":"pone.0314858.ref002","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1186\/s13023-018-0872-9","article-title":"A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life","volume":"13","author":"N Bascou","year":"2018","journal-title":"Orphanet J Rare Dis"},{"issue":"3","key":"pone.0314858.ref003","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1016\/j.pediatrneurol.2011.05.007","article-title":"Early infantile Krabbe disease: results of the World-Wide Krabbe Registry","volume":"45","author":"PK Duffner","year":"2011","journal-title":"Pediatr Neurol"},{"issue":"5","key":"pone.0314858.ref004","doi-asserted-by":"crossref","first-page":"298","DOI":"10.1016\/j.pediatrneurol.2012.02.023","article-title":"Later onset phenotypes of Krabbe disease: results of the world-wide registry","volume":"46","author":"PK Duffner","year":"2012","journal-title":"Pediatr Neurol"},{"key":"pone.0314858.ref005","article-title":"Oligodendrocytes: Myelination, Plasticity, and Axonal Support","author":"M Simons","year":"2024","journal-title":"Cold Spring Harb Perspect Biol"},{"key":"pone.0314858.ref006","doi-asserted-by":"crossref","first-page":"145","DOI":"10.3389\/fnins.2014.00145","article-title":"Oligodendrogenesis in the normal and pathological central nervous system","volume":"8","author":"B El Waly","year":"2014","journal-title":"Front Neurosci"},{"key":"pone.0314858.ref007","article-title":"Microglia in Health and Diseases: Integrative Hubs of the Central Nervous System (CNS)","author":"A Sierra","year":"2024","journal-title":"Cold Spring Harb Perspect Biol"},{"issue":"2","key":"pone.0314858.ref008","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1016\/0006-8993(80)90159-6","article-title":"The Twitcher mouse: an enzymatically authentic model of human globoid cell leukodystrophy (Krabbe disease)","volume":"202","author":"T Kobayashi","year":"1980","journal-title":"Brain Res"},{"issue":"1","key":"pone.0314858.ref009","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1016\/j.neuron.2020.03.031","article-title":"Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction","volume":"107","author":"NI Weinstock","year":"2020","journal-title":"Neuron"},{"issue":"6","key":"pone.0314858.ref010","doi-asserted-by":"crossref","first-page":"1858","DOI":"10.1523\/JNEUROSCI.3095-15.2016","article-title":"Altered Trafficking and Processing of GALC Mutants Correlates with Globoid Cell Leukodystrophy Severity","volume":"36","author":"D Shin","year":"2016","journal-title":"J Neurosci"},{"issue":"40","key":"pone.0314858.ref011","doi-asserted-by":"crossref","first-page":"20097","DOI":"10.1073\/pnas.1912108116","article-title":"Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target","volume":"116","author":"Y Li","year":"2019","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"4","key":"pone.0314858.ref012","doi-asserted-by":"crossref","first-page":"604","DOI":"10.1042\/bj0600604","article-title":"Tissue fractionation studies. 6. 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Wright","year":"2017","journal-title":"Neurology"},{"issue":"1","key":"pone.0314858.ref038","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1016\/j.ymthe.2022.09.017","article-title":"Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases","volume":"31","author":"G Heller","year":"2023","journal-title":"Mol Ther"},{"issue":"9\u201310","key":"pone.0314858.ref039","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1089\/hum.2021.245","article-title":"Efficacy and Safety of a Krabbe Disease Gene Therapy","volume":"33","author":"J Hordeaux","year":"2022","journal-title":"Hum Gene Ther"},{"issue":"2","key":"pone.0314858.ref040","doi-asserted-by":"crossref","first-page":"135","DOI":"10.34172\/bi.2021.21","article-title":"Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?","volume":"11","author":"MA Rafi","year":"2021","journal-title":"Bioimpacts"},{"issue":"11","key":"pone.0314858.ref041","doi-asserted-by":"crossref","first-page":"1681","DOI":"10.1038\/mt.2015.145","article-title":"Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice","volume":"23","author":"MA Rafi","year":"2015","journal-title":"Mol Ther"},{"issue":"2","key":"pone.0314858.ref042","doi-asserted-by":"crossref","first-page":"691","DOI":"10.1016\/j.ymthe.2020.12.031","article-title":"Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease","volume":"29","author":"Y Li","year":"2021","journal-title":"Mol Ther"},{"issue":"1","key":"pone.0314858.ref043","doi-asserted-by":"crossref","first-page":"210","DOI":"10.1186\/s12864-023-09285-6","article-title":"Identifying altered developmental pathways in human globoid cell leukodystrophy iPSCs-derived NSCs using transcriptome 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