Patient-Specific Midbrain Organoids with CRISPR Correction Reveal Disease Mechanisms and Enable Therapeutic Evaluation in Neuronopathic Gaucher Disease
利用 CRISPR 技术进行基因编辑的患者特异性中脑类器官揭示了神经病变型戈谢病的疾病机制,并可用于治疗评估。
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影响因子:
doi:10.1101/2025.11.06.686937
Lin, Yi; Liou, Benjamin; Fannin, Venette; Adler, Stuart; Mayhew, Christopher N; Hammonds, Jason E; Hu, Yueh-Chiang; Tchieu, Jason; Zhang, Wujuan; Zhao, Xueheng; Beres, Rebecca L; Setchell, Kenneth Dr; Kaynak, Ahmet; Qi, Xiaoyang; Feldman, Ricardo A; Sun, Ying