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 H. Tchieu, Wujuan Zhang, Xueheng Zhao, Rebecca L. Beres, Kenneth D.R. Setchell, Ahmet Kaynak, Xiaoyang Qi, Ricardo A. Feldman, Ying Sun
This paper develops patient-specific midbrain organoids with CRISPR correction to model neuronopathic Gaucher disease and test therapies.
bioRxiv · Nov 6, 2025Neuroscience