Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies

Neuronopathic Gaucher disease (nGD) is a lysosomal storage disorder caused by GBA1 mutations, leading to defective acid β-glucosidase (GCase) and accumulation of glycosphingolipid substrates, causing inflammation and neurodegeneration. Patients with nGD manifest severe neurological symptoms, but cur...

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Hlavní autoři:	Yi Lin, Benjamin Liou, Venette Fannin, Stuart Adler, Christopher N Mayhew, Jason E Hammonds, Yueh-Chiang Hu, Jason Tchieu, Wujuan Zhang, Xueheng Zhao, Rebecca L Beres, Kenneth DR Setchell, Ahmet Kaynak, Xiaoyang Qi, Ricardo A Feldman, Ying Sun
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	eLife Sciences Publications Ltd 2026-06-01
Edice:	eLife
Témata:	neuronopathic Gaucher disease midbrain-like organoids iPSC disease modeling dopaminergic neurons lipid metabolism gene editing
On-line přístup:	https://elifesciences.org/articles/109518
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