Restoration of Progranulin Expression Rescues Cortical Neuron Generation in an Induced Pluripotent Stem Cell Model of Frontotemporal Dementia
Identifieur interne : 000218 ( Main/Exploration ); précédent : 000217; suivant : 000219Restoration of Progranulin Expression Rescues Cortical Neuron Generation in an Induced Pluripotent Stem Cell Model of Frontotemporal Dementia
Auteurs : Susanna Raitano [Belgique] ; Laura Ordovàs [Belgique] ; Louis De Muynck [Belgique] ; Wenting Guo [Belgique] ; Ira Espuny-Camacho [Belgique] ; Martine Geraerts [Belgique] ; Satish Khurana [Belgique] ; Kim Vanuytsel [Belgique] ; Balazs I. T Th [Belgique] ; Thomas Voets [Belgique] ; Rik Vandenberghe [Belgique] ; Toni Cathomen [Allemagne] ; Ludo Van Den Bosch [Belgique] ; Pierre Vanderhaeghen [Belgique] ; Philip Van Damme [Belgique] ; Catherine M. Verfaillie [Belgique]Source :
- Stem Cell Reports [ 2213-6711 ] ; 2014.
Abstract
To understand how haploinsufficiency of progranulin (PGRN) causes frontotemporal dementia (FTD), we created induced pluripotent stem cells (iPSCs) from patients carrying the
Url:
DOI: 10.1016/j.stemcr.2014.12.001
PubMed: 25556567
PubMed Central: 4297877
Affiliations:
- Allemagne, Belgique
- Bade-Wurtemberg, District de Fribourg-en-Brisgau, Province du Brabant flamand, Vienne (Autriche)
- Fribourg-en-Brisgau, Louvain, Vienne (Autriche)
- Université libre de Bruxelles
Links toward previous steps (curation, corpus...)
- to stream Pmc, to step Corpus: 000261
- to stream Pmc, to step Curation: 000261
- to stream Pmc, to step Checkpoint: 000183
- to stream Ncbi, to step Merge: 000284
- to stream Ncbi, to step Curation: 000284
- to stream Ncbi, to step Checkpoint: 000284
- to stream Main, to step Merge: 000218
- to stream Main, to step Curation: 000218
Le document en format XML
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Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Vanuytsel, Kim" sort="Vanuytsel, Kim" uniqKey="Vanuytsel K" first="Kim" last="Vanuytsel">Kim Vanuytsel</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="T Th, Balazs I" sort="T Th, Balazs I" uniqKey="T Th B" first="Balazs I." last="T Th">Balazs I. T Th</name><affiliation wicri:level="3"><nlm:aff id="aff10">Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Voets, Thomas" sort="Voets, Thomas" uniqKey="Voets T" first="Thomas" last="Voets">Thomas Voets</name><affiliation wicri:level="3"><nlm:aff id="aff10">Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Vandenberghe, Rik" sort="Vandenberghe, Rik" uniqKey="Vandenberghe R" first="Rik" last="Vandenberghe">Rik Vandenberghe</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant 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nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Fribourg-en-Brisgau</region><settlement type="city">Fribourg-en-Brisgau</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff12">Center for Chronic Immunodeficiency, University Medical Center, 79108 Freiburg, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Center for Chronic Immunodeficiency, University Medical Center, 79108 Freiburg</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Fribourg-en-Brisgau</region><settlement type="city">Fribourg-en-Brisgau</settlement></placeName></affiliation></author><author><name sortKey="Van Den Bosch, Ludo" sort="Van Den Bosch, Ludo" uniqKey="Van Den Bosch L" first="Ludo" last="Van Den Bosch">Ludo Van Den Bosch</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff5">Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><affiliation wicri:level="4"><nlm:aff id="aff6">Institute for Interdisciplinary Research (IRIBHM), Université Libre de Bruxelles (ULB), 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Institute for Interdisciplinary Research (IRIBHM), Université Libre de Bruxelles (ULB), 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName><orgName type="university">Université libre de Bruxelles</orgName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff7">Institute of Neuroscience (UNI), ULB, 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Institute of Neuroscience (UNI), ULB, 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff8">VIB Center for the Biology of Disease, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>VIB Center for the Biology of Disease, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff9">Center of Human Genetics, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Center of Human Genetics, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff13">Walloon Excellence in Life Sciences and Biotechnology (WELBIO), 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Walloon Excellence in Life Sciences and Biotechnology (WELBIO), 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName></affiliation></author><author><name sortKey="Van Damme, Philip" sort="Van Damme, Philip" uniqKey="Van Damme P" first="Philip" last="Van Damme">Philip Van Damme</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff5">Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff14">Department of Neurology, University Hospitals Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Department of Neurology, University Hospitals Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Verfaillie, Catherine M" sort="Verfaillie, Catherine M" uniqKey="Verfaillie C" first="Catherine M." last="Verfaillie">Catherine M. Verfaillie</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">25556567</idno><idno type="pmc">4297877</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4297877</idno><idno type="RBID">PMC:4297877</idno><idno type="doi">10.1016/j.stemcr.2014.12.001</idno><date when="2014">2014</date><idno type="wicri:Area/Pmc/Corpus">000261</idno><idno type="wicri:Area/Pmc/Curation">000261</idno><idno type="wicri:Area/Pmc/Checkpoint">000183</idno><idno type="wicri:Area/Ncbi/Merge">000284</idno><idno type="wicri:Area/Ncbi/Curation">000284</idno><idno type="wicri:Area/Ncbi/Checkpoint">000284</idno><idno type="wicri:Area/Main/Merge">000218</idno><idno type="wicri:Area/Main/Curation">000218</idno><idno type="wicri:Area/Main/Exploration">000218</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Restoration of Progranulin Expression Rescues Cortical Neuron Generation in an Induced Pluripotent Stem Cell Model of Frontotemporal Dementia</title><author><name sortKey="Raitano, Susanna" sort="Raitano, Susanna" uniqKey="Raitano S" first="Susanna" last="Raitano">Susanna Raitano</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Ordovas, Laura" sort="Ordovas, Laura" uniqKey="Ordovas L" first="Laura" last="Ordovàs">Laura Ordovàs</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="De Muynck, Louis" sort="De Muynck, Louis" uniqKey="De Muynck L" first="Louis" last="De Muynck">Louis De Muynck</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff5">Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Guo, Wenting" sort="Guo, Wenting" uniqKey="Guo W" first="Wenting" last="Guo">Wenting Guo</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff5">Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Espuny Camacho, Ira" sort="Espuny Camacho, Ira" uniqKey="Espuny Camacho I" first="Ira" last="Espuny-Camacho">Ira Espuny-Camacho</name><affiliation wicri:level="4"><nlm:aff id="aff6">Institute for Interdisciplinary Research (IRIBHM), Université Libre de Bruxelles (ULB), 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Institute for Interdisciplinary Research (IRIBHM), Université Libre de Bruxelles (ULB), 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName><orgName type="university">Université libre de Bruxelles</orgName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff7">Institute of Neuroscience (UNI), ULB, 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Institute of Neuroscience (UNI), ULB, 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff8">VIB Center for the Biology of Disease, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>VIB Center for the Biology of Disease, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff9">Center of Human Genetics, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Center of Human Genetics, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Geraerts, Martine" sort="Geraerts, Martine" uniqKey="Geraerts M" first="Martine" last="Geraerts">Martine Geraerts</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Khurana, Satish" sort="Khurana, Satish" uniqKey="Khurana S" first="Satish" last="Khurana">Satish Khurana</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Vanuytsel, Kim" sort="Vanuytsel, Kim" uniqKey="Vanuytsel K" first="Kim" last="Vanuytsel">Kim Vanuytsel</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="T Th, Balazs I" sort="T Th, Balazs I" uniqKey="T Th B" first="Balazs I." last="T Th">Balazs I. T Th</name><affiliation wicri:level="3"><nlm:aff id="aff10">Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Voets, Thomas" sort="Voets, Thomas" uniqKey="Voets T" first="Thomas" last="Voets">Thomas Voets</name><affiliation wicri:level="3"><nlm:aff id="aff10">Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Ion Channel Research, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Vandenberghe, Rik" sort="Vandenberghe, Rik" uniqKey="Vandenberghe R" first="Rik" last="Vandenberghe">Rik Vandenberghe</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff14">Department of Neurology, University Hospitals Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Department of Neurology, University Hospitals Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Cathomen, Toni" sort="Cathomen, Toni" uniqKey="Cathomen T" first="Toni" last="Cathomen">Toni Cathomen</name><affiliation wicri:level="3"><nlm:aff id="aff11">Institute for Cell and Gene Therapy, University Medical Center, 79108 Freiburg, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Institute for Cell and Gene Therapy, University Medical Center, 79108 Freiburg</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Fribourg-en-Brisgau</region><settlement type="city">Fribourg-en-Brisgau</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff12">Center for Chronic Immunodeficiency, University Medical Center, 79108 Freiburg, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Center for Chronic Immunodeficiency, University Medical Center, 79108 Freiburg</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Fribourg-en-Brisgau</region><settlement type="city">Fribourg-en-Brisgau</settlement></placeName></affiliation></author><author><name sortKey="Van Den Bosch, Ludo" sort="Van Den Bosch, Ludo" uniqKey="Van Den Bosch L" first="Ludo" last="Van Den Bosch">Ludo Van Den Bosch</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff5">Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><affiliation wicri:level="4"><nlm:aff id="aff6">Institute for Interdisciplinary Research (IRIBHM), Université Libre de Bruxelles (ULB), 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Institute for Interdisciplinary Research (IRIBHM), Université Libre de Bruxelles (ULB), 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName><orgName type="university">Université libre de Bruxelles</orgName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff7">Institute of Neuroscience (UNI), ULB, 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Institute of Neuroscience (UNI), ULB, 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff8">VIB Center for the Biology of Disease, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>VIB Center for the Biology of Disease, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff9">Center of Human Genetics, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Center of Human Genetics, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff13">Walloon Excellence in Life Sciences and Biotechnology (WELBIO), 1070 Brussels, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Walloon Excellence in Life Sciences and Biotechnology (WELBIO), 1070 Brussels</wicri:regionArea><placeName><region type="land" nuts="2">Vienne (Autriche)</region><settlement type="city">Vienne (Autriche)</settlement></placeName></affiliation></author><author><name sortKey="Van Damme, Philip" sort="Van Damme, Philip" uniqKey="Van Damme P" first="Philip" last="Van Damme">Philip Van Damme</name><affiliation wicri:level="3"><nlm:aff id="aff3">Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven Research Institute for Neuroscience and Disease (LIND), KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff4">Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Research Group Experimental Neurology, Department of Neurosciences, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff5">Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Laboratory of Neurobiology, VIB Vesalius Research Center, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff14">Department of Neurology, University Hospitals Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Department of Neurology, University Hospitals Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author><author><name sortKey="Verfaillie, Catherine M" sort="Verfaillie, Catherine M" uniqKey="Verfaillie C" first="Catherine M." last="Verfaillie">Catherine M. Verfaillie</name><affiliation wicri:level="3"><nlm:aff id="aff1">Stem Cell Institute, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Institute, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation><affiliation wicri:level="3"><nlm:aff id="aff2">Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium</nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Stem Cell Biology and Embryology Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven</wicri:regionArea><placeName><region type="province" nuts="2">Province du Brabant flamand</region><settlement type="city">Louvain</settlement></placeName></affiliation></author></analytic><series><title level="j">Stem Cell Reports</title><idno type="eISSN">2213-6711</idno><imprint><date when="2014">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><title>Summary</title><p>To understand how haploinsufficiency of progranulin (PGRN) causes frontotemporal dementia (FTD), we created induced pluripotent stem cells (iPSCs) from patients carrying the <italic>GRN</italic><sup>IVS1+5G > C</sup> mutation (FTD-iPSCs). FTD-iPSCs were fated to cortical neurons, the cells most affected in FTD. Although generation of neuroprogenitors was unaffected, their further differentiation into CTIP2-, FOXP2-, or TBR1-TUJ1 double-positive cortical neurons, but not motorneurons, was significantly decreased in FTD-neural progeny. Zinc finger nuclease-mediated introduction of <italic>GRN</italic> cDNA into the <italic>AAVS1</italic> locus corrected defects in cortical neurogenesis, demonstrating that PGRN haploinsufficiency causes inefficient cortical neuron generation. RNA sequencing analysis confirmed reversal of the altered gene expression profile following genetic correction. We identified the Wnt signaling pathway as one of the top defective pathways in FTD-iPSC-derived neurons, which was reversed following genetic correction. Differentiation of FTD-iPSCs in the presence of a WNT inhibitor mitigated defective corticogenesis. Therefore, we demonstrate that PGRN haploinsufficiency hampers corticogenesis in vitro.</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Almeida, S" uniqKey="Almeida S">S. Almeida</name></author><author><name sortKey="Zhang, Z" uniqKey="Zhang Z">Z. Zhang</name></author><author><name sortKey="Coppola, G" uniqKey="Coppola G">G. Coppola</name></author><author><name sortKey="Mao, W" uniqKey="Mao W">W. Mao</name></author><author><name sortKey="Futai, K" uniqKey="Futai K">K. Futai</name></author><author><name sortKey="Karydas, A" uniqKey="Karydas A">A. Karydas</name></author><author><name sortKey="Geschwind, M D" uniqKey="Geschwind M">M.D. Geschwind</name></author><author><name sortKey="Tartaglia, M C" uniqKey="Tartaglia M">M.C. Tartaglia</name></author><author><name sortKey="Gao, F" uniqKey="Gao F">F. Gao</name></author><author><name sortKey="Gianni, D" uniqKey="Gianni D">D. 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Palmer</name></author><author><name sortKey="Geschwind, D H" uniqKey="Geschwind D">D.H. Geschwind</name></author></analytic></biblStruct></listBibl></div1></back></TEI><affiliations><list><country><li>Allemagne</li><li>Belgique</li></country><region><li>Bade-Wurtemberg</li><li>District de Fribourg-en-Brisgau</li><li>Province du Brabant flamand</li><li>Vienne (Autriche)</li></region><settlement><li>Fribourg-en-Brisgau</li><li>Louvain</li><li>Vienne (Autriche)</li></settlement><orgName><li>Université libre de Bruxelles</li></orgName></list><tree><country name="Belgique"><region name="Province du Brabant flamand"><name sortKey="Raitano, Susanna" sort="Raitano, Susanna" uniqKey="Raitano S" first="Susanna" last="Raitano">Susanna Raitano</name></region><name sortKey="De Muynck, Louis" sort="De Muynck, Louis" uniqKey="De Muynck L" first="Louis" last="De Muynck">Louis De Muynck</name><name sortKey="De Muynck, Louis" sort="De Muynck, Louis" uniqKey="De Muynck L" first="Louis" last="De Muynck">Louis De Muynck</name><name sortKey="De Muynck, Louis" sort="De Muynck, Louis" uniqKey="De Muynck L" first="Louis" last="De Muynck">Louis De Muynck</name><name sortKey="Espuny Camacho, Ira" sort="Espuny Camacho, Ira" uniqKey="Espuny Camacho I" first="Ira" last="Espuny-Camacho">Ira Espuny-Camacho</name><name sortKey="Espuny Camacho, Ira" sort="Espuny Camacho, Ira" uniqKey="Espuny Camacho I" first="Ira" last="Espuny-Camacho">Ira Espuny-Camacho</name><name sortKey="Espuny Camacho, Ira" sort="Espuny Camacho, Ira" uniqKey="Espuny Camacho I" first="Ira" last="Espuny-Camacho">Ira Espuny-Camacho</name><name sortKey="Espuny Camacho, Ira" sort="Espuny Camacho, Ira" uniqKey="Espuny Camacho I" first="Ira" last="Espuny-Camacho">Ira Espuny-Camacho</name><name sortKey="Geraerts, Martine" sort="Geraerts, Martine" uniqKey="Geraerts M" first="Martine" last="Geraerts">Martine Geraerts</name><name sortKey="Geraerts, Martine" sort="Geraerts, Martine" uniqKey="Geraerts M" first="Martine" last="Geraerts">Martine Geraerts</name><name sortKey="Guo, Wenting" sort="Guo, Wenting" uniqKey="Guo W" first="Wenting" last="Guo">Wenting Guo</name><name sortKey="Guo, Wenting" sort="Guo, Wenting" uniqKey="Guo W" first="Wenting" last="Guo">Wenting Guo</name><name sortKey="Guo, Wenting" sort="Guo, Wenting" uniqKey="Guo W" first="Wenting" last="Guo">Wenting Guo</name><name sortKey="Khurana, Satish" sort="Khurana, Satish" uniqKey="Khurana S" first="Satish" last="Khurana">Satish Khurana</name><name sortKey="Khurana, Satish" sort="Khurana, Satish" uniqKey="Khurana S" first="Satish" last="Khurana">Satish Khurana</name><name sortKey="Ordovas, Laura" sort="Ordovas, Laura" uniqKey="Ordovas L" first="Laura" last="Ordovàs">Laura Ordovàs</name><name sortKey="Ordovas, Laura" sort="Ordovas, Laura" uniqKey="Ordovas L" first="Laura" last="Ordovàs">Laura Ordovàs</name><name sortKey="Raitano, Susanna" sort="Raitano, Susanna" uniqKey="Raitano S" first="Susanna" last="Raitano">Susanna Raitano</name><name sortKey="T Th, Balazs I" sort="T Th, Balazs I" uniqKey="T Th B" first="Balazs I." last="T Th">Balazs I. T Th</name><name sortKey="Van Damme, Philip" sort="Van Damme, Philip" uniqKey="Van Damme P" first="Philip" last="Van Damme">Philip Van Damme</name><name sortKey="Van Damme, Philip" sort="Van Damme, Philip" uniqKey="Van Damme P" first="Philip" last="Van Damme">Philip Van Damme</name><name sortKey="Van Damme, Philip" sort="Van Damme, Philip" uniqKey="Van Damme P" first="Philip" last="Van Damme">Philip Van Damme</name><name sortKey="Van Damme, Philip" sort="Van Damme, Philip" uniqKey="Van Damme P" first="Philip" last="Van Damme">Philip Van Damme</name><name sortKey="Van Den Bosch, Ludo" sort="Van Den Bosch, Ludo" uniqKey="Van Den Bosch L" first="Ludo" last="Van Den Bosch">Ludo Van Den Bosch</name><name sortKey="Van Den Bosch, Ludo" sort="Van Den Bosch, Ludo" uniqKey="Van Den Bosch L" first="Ludo" last="Van Den Bosch">Ludo Van Den Bosch</name><name sortKey="Van Den Bosch, Ludo" sort="Van Den Bosch, Ludo" uniqKey="Van Den Bosch L" first="Ludo" last="Van Den Bosch">Ludo Van Den Bosch</name><name sortKey="Vandenberghe, Rik" sort="Vandenberghe, Rik" uniqKey="Vandenberghe R" first="Rik" last="Vandenberghe">Rik Vandenberghe</name><name sortKey="Vandenberghe, Rik" sort="Vandenberghe, Rik" uniqKey="Vandenberghe R" first="Rik" last="Vandenberghe">Rik Vandenberghe</name><name sortKey="Vandenberghe, Rik" sort="Vandenberghe, Rik" uniqKey="Vandenberghe R" first="Rik" last="Vandenberghe">Rik Vandenberghe</name><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><name sortKey="Vanderhaeghen, Pierre" sort="Vanderhaeghen, Pierre" uniqKey="Vanderhaeghen P" first="Pierre" last="Vanderhaeghen">Pierre Vanderhaeghen</name><name sortKey="Vanuytsel, Kim" sort="Vanuytsel, Kim" uniqKey="Vanuytsel K" first="Kim" last="Vanuytsel">Kim Vanuytsel</name><name sortKey="Vanuytsel, Kim" sort="Vanuytsel, Kim" uniqKey="Vanuytsel K" first="Kim" last="Vanuytsel">Kim Vanuytsel</name><name sortKey="Verfaillie, Catherine M" sort="Verfaillie, Catherine M" uniqKey="Verfaillie C" first="Catherine M." last="Verfaillie">Catherine M. Verfaillie</name><name sortKey="Verfaillie, Catherine M" sort="Verfaillie, Catherine M" uniqKey="Verfaillie C" first="Catherine M." last="Verfaillie">Catherine M. Verfaillie</name><name sortKey="Voets, Thomas" sort="Voets, Thomas" uniqKey="Voets T" first="Thomas" last="Voets">Thomas Voets</name></country><country name="Allemagne"><region name="Bade-Wurtemberg"><name sortKey="Cathomen, Toni" sort="Cathomen, Toni" uniqKey="Cathomen T" first="Toni" last="Cathomen">Toni Cathomen</name></region><name sortKey="Cathomen, Toni" sort="Cathomen, Toni" uniqKey="Cathomen T" first="Toni" last="Cathomen">Toni Cathomen</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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