ArticleeLife2026
Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.
Yi Lin, Benjamin Liou, Venette Fannin, Stuart Adler, Christopher N Mayhew, Jason E Hammonds, Yueh-Chiang Hu, Jason Tchieu, Wujuan Zhang, Xueheng Zhao and 6 more
Abstract read
In one paragraphArticle in eLife, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
2 · The registryThe trial behind it
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3 · Its place in the literatureWho cites it
0 citing papers in PubMed.
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4 · The recordCorrections and comments
5 · Who and what moneyAuthors and funding
16 authors.
Venette FanninDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, United States.
Stuart AdlerDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, United States.
Christopher N MayhewDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, United States.
Jason E HammondsDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, United States.
Yueh-Chiang HuDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, United States.
Wujuan ZhangDivision of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, United States.
Xueheng ZhaoDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, United States.
Rebecca L BeresDivision of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, United States.
Kenneth D R SetchellDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, United States.
Funding
Progranulin: A Novel Gene in Gaucher DiseasesR01NS103931 · NINDS · YALE UNIVERSITY · PI Chuanju Liu, Ying Sun · 2017 to 2026
$4.3MSystemic Enzyme Delivery by a Brain Targeted SapC-DOPS Nanocarrier for Treatment of Neuronopathic Gaucher DiseaseR01NS138309 · NINDS · CINCINNATI CHILDRENS HOSP MED CTR · PI XIAOYANG QI, Ying Sun · 2024 to 2026
$1.9MBrain organoid modeling of Gaucher disease for identification of pathogenic pathways and therapy developmentR21HD102788 · NICHD · CINCINNATI CHILDRENS HOSP MED CTR · PI SUN, YING · 2020 to 2021
$437kCincinnati Children's Hospital Medical Center Center of Pediatric Genomics AwardCincinnati Children's Hospital Medical Center Research Innovation Pilot Funding Program AwardNational Institute of Health R01NS103931National Institute of Health R21OD033660NICHD NIH HHS R21 HD102788NICHD NIH HHS R21HD1027881NIH HHS 2UL1TR001425-05A1, CHMC-CTSA 00003827NINDS NIH HHS R01 NS103931NINDS NIH HHS R01 NS138309NINDS NIH HHS R01NS138309
6 · The paper itselfAbstract
Neuronopathic Gaucher disease (nGD) is a lysosomal storage disorder caused by
Indexed as
CRISPR-Cas SystemsGaucher DiseaseGlucosylceramidaseMesencephalonOrganoidsDopaminergic NeuronsHumansInduced Pluripotent Stem CellsMutationPhenotypeGBA protein, humanGlucosylceramidasedopaminergic neuronsgene editinghumaniPSC disease modelinglipid metabolismmidbrain-like organoidsneuronopathic Gaucher diseaseneuroscienceregenerative medicinestem cells
Identifiers
PMID42334452
PMCPMC13290227
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