{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,14]],"date-time":"2026-04-14T20:52:21Z","timestamp":1776199941100,"version":"3.50.1"},"reference-count":45,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2020,12,30]],"date-time":"2020-12-30T00:00:00Z","timestamp":1609286400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100002661","name":"Fonds De La Recherche Scientifique - FNRS","doi-asserted-by":"publisher","award":["FRIA Grant"],"award-info":[{"award-number":["FRIA Grant"]}],"id":[{"id":"10.13039\/501100002661","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cells"],"abstract":"<jats:p>Genetic alterations in PEX genes lead to peroxisome biogenesis disorder. In humans, they are associated with Zellweger spectrum disorders (ZSD). No validated treatment has been shown to modify the dismal natural history of ZSD. Liver transplantation (LT) improved clinical and biochemical outcomes in mild ZSD patients. Hepatocyte transplantation (HT), developed to overcome LT limitations, was performed in a mild ZSD 4-year-old child with encouraging short-term results. Here, we evaluated low dose (12.5 million hepatocytes\/kg) and high dose (50 million hepatocytes\/kg) syngeneic male HT via intrasplenic infusion in the Pex1-G844D NMRI mouse model which recapitulates a mild ZSD phenotype. HT was feasible and safe in growth retarded ZSD mice. Clinical (weight and food intake) and biochemical parameters (very long-chain fatty acids, abnormal bile acids, etc.) were in accordance with ZSD phenotype but they were not robustly modified by HT. As expected, one third of the infused cells were detected in the liver 24 h post-HT. No liver nor spleen microchimerism was detected after 7, 14 and 30 days. Future optimizations are required to improve hepatocyte engraftment in Pex1-G844D NMRI mouse liver. The mouse model exhibited the robustness required for ZSD liver-targeted therapies evaluation.<\/jats:p>","DOI":"10.3390\/cells10010040","type":"journal-article","created":{"date-parts":[[2020,12,30]],"date-time":"2020-12-30T09:35:23Z","timestamp":1609320923000},"page":"40","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["High Dose Versus Low Dose Syngeneic Hepatocyte Transplantation in Pex1-G844D NMRI Mouse Model is Safe but Does Not Achieve Long Term Engraftment"],"prefix":"10.3390","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7605-9031","authenticated-orcid":false,"given":"Tanguy","family":"Demaret","sequence":"first","affiliation":[{"name":"Laboratoire d\u2019H\u00e9patologie P\u00e9diatrique et Th\u00e9rapie Cellulaire, Unit\u00e9 PEDI, Institut de Recherche Exp\u00e9rimentale et Clinique (IREC), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan","family":"Evraerts","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019H\u00e9patologie P\u00e9diatrique et Th\u00e9rapie Cellulaire, Unit\u00e9 PEDI, Institut de Recherche Exp\u00e9rimentale et Clinique (IREC), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joachim","family":"Ravau","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019H\u00e9patologie P\u00e9diatrique et Th\u00e9rapie Cellulaire, Unit\u00e9 PEDI, Institut de Recherche Exp\u00e9rimentale et Clinique (IREC), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8915-795X","authenticated-orcid":false,"given":"Martin","family":"Roumain","sequence":"additional","affiliation":[{"name":"Bioanalysis and Pharmacology of Bioactive Lipids Research Group (BPBL), Louvain Drug Research Institute (LDRI), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1600-9259","authenticated-orcid":false,"given":"Giulio G.","family":"Muccioli","sequence":"additional","affiliation":[{"name":"Bioanalysis and Pharmacology of Bioactive Lipids Research Group (BPBL), Louvain Drug Research Institute (LDRI), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7329-9635","authenticated-orcid":false,"given":"Mustapha","family":"Najimi","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019H\u00e9patologie P\u00e9diatrique et Th\u00e9rapie Cellulaire, Unit\u00e9 PEDI, Institut de Recherche Exp\u00e9rimentale et Clinique (IREC), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5597-4708","authenticated-orcid":false,"given":"Etienne M.","family":"Sokal","sequence":"additional","affiliation":[{"name":"Laboratoire d\u2019H\u00e9patologie P\u00e9diatrique et Th\u00e9rapie Cellulaire, Unit\u00e9 PEDI, Institut de Recherche Exp\u00e9rimentale et Clinique (IREC), Universit\u00e9 Catholique de Louvain (UCLouvain), 1200 Brussels, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2020,12,30]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1016\/j.ymgme.2004.08.008","article-title":"The PEX Gene Screen: Molecular diagnosis of peroxisome biogenesis disorders in the Zellweger syndrome spectrum","volume":"83","author":"Steinberg","year":"2004","journal-title":"Mol. 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