Intravenous infusion of iPSC-derived neural precursor cells increases acid β-glucosidase function in the brain and lessens the neuronopathic phenotype in a mouse model of Gaucher disease

Gaucher disease (GD) is caused by GBA1 mutations leading to functional deficiency of acid-β-glucosidase (GCase). No effective treatment is available for neuronopathic GD (nGD). A subclass of neural stem and precursor cells (NPCs) expresses VLA4 (integrin α4β1, very late antigen-4) that facilitates N...

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Veröffentlicht in:Hum Mol Genet
Hauptverfasser: Peng, Yanyan, Liou, Benjamin, Inskeep, Venette, Blackwood, Rachel, Mayhew, Christopher N, Grabowski, Gregory A, Sun, Ying
Format: Artigo
Sprache:Inglês
Veröffentlicht: Oxford University Press 2019
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General Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6891072/
https://ncbi.nlm.nih.gov/pubmed/31373366
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz184
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