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No effective treatment is available for neuronopathic GD (nGD). A subclass of neural stem and precursor cells (NPCs) expresses VLA4 (integrin \u03b14\u03b21, very late antigen-4) that facilitates NPC entry into the brain following intravenous (IV) infusion. Here, the therapeutic potential of IV VLA4+NPCs was assessed for nGD using wild-type mouse green fluorescent protein (GFP)-positive multipotent induced pluripotent stem cell (iPSC)-derived VLA4+NPCs. VLA4+NPCs successfully engrafted in the nGD (4L;C*) mouse brain. GFP-positive cells differentiated into neurons, astrocytes and oligodendrocytes in the brainstem, midbrain and thalamus of the transplanted mice and significantly improved sensorimotor function and prolonged life span compared to vehicle-treated 4L;C* mice. VLA4+NPC transplantation significantly decreased levels of CD68 and glial fibrillary acidic protein, as well as TNF\u03b1 mRNA levels in the brain, indicating reduced neuroinflammation. Furthermore, decreased Fluoro-Jade C and NeuroSilver staining suggested inhibition of neurodegeneration. VLA4+NPC-engrafted 4L;C* midbrains showed 35% increased GCase activity, reduced substrate [glucosylceramide (GC, \u221234%) and glucosylsphingosine (GS, \u221211%)] levels and improved mitochondrial oxygen consumption rates in comparison to vehicle-4L;C* mice. VLA4+NPC engraftment in 4L;C* brain also led to enhanced expression of neurotrophic factors that have roles in neuronal survival and the promotion of neurogenesis. This study provides evidence that iPSC-derived NPC transplantation has efficacy in an nGD mouse model and provides proof of concept for autologous NPC therapy in nGD.<\/jats:p>","DOI":"10.1093\/hmg\/ddz184","type":"journal-article","created":{"date-parts":[[2019,7,24]],"date-time":"2019-07-24T07:12:03Z","timestamp":1563952323000},"page":"3406-3421","source":"Crossref","is-referenced-by-count":12,"title":["Intravenous infusion of iPSC-derived neural precursor cells increases acid \u03b2-glucosidase function in the brain and lessens the neuronopathic phenotype in a mouse model of Gaucher disease"],"prefix":"10.1093","volume":"28","author":[{"given":"Yanyan","family":"Peng","sequence":"first","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"Liou","sequence":"additional","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Venette","family":"Inskeep","sequence":"additional","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel","family":"Blackwood","sequence":"additional","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher N","family":"Mayhew","sequence":"additional","affiliation":[{"name":"Division of Developmental Biology, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"},{"name":"Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory A","family":"Grabowski","sequence":"additional","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ying","family":"Sun","sequence":"additional","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH 45229, USA"},{"name":"Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2019,8,2]]},"reference":[{"key":"2019120404004523800_ref1","volume-title":"The Online Metabolic and Molecular Bases of Inherited Diseases","author":"Grabowski","year":"2010"},{"issue":"Suppl 1","key":"2019120404004523800_ref2","doi-asserted-by":"crossref","first-page":"S12","DOI":"10.1002\/ajh.24063","article-title":"Gaucher disease types 1 and 3: phenotypic characterization of large populations from the ICGG Gaucher registry","volume":"90","author":"Grabowski","year":"2015","journal-title":"Am. 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Hematol."},{"key":"2019120404004523800_ref3","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1086\/302925","article-title":"Analysis and classification of 304 mutant alleles in patients with type 1 and type 3 Gaucher disease","volume":"66","author":"Koprivica","year":"2000","journal-title":"Am J Hum Genet"},{"key":"2019120404004523800_ref4","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1016\/j.bcmd.2006.11.003","article-title":"Gaucher disease among Chinese patients: review on genotype\/phenotype correlation from 29 patients and identification of novel and rare alleles","volume":"38","author":"Choy","year":"2007","journal-title":"Blood Cells Mol Dis"},{"key":"2019120404004523800_ref5","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1016\/j.bcmd.2010.07.010","article-title":"Clinical and genetic characteristics of Korean patients with Gaucher disease","volume":"46","author":"Jeong","year":"2011","journal-title":"Blood Cells Mol 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