基于iPSC的自体细胞治疗
(Shi, Y., et al. Nat Rev Drug Discov, 2017)
基于iPSC的自体细胞治疗
iPSC具有无限扩增的能力,并且可以分化为成体所有细胞类型,可为再生医学提供无限细胞来源。结合基因修饰技术,我们可以在获得病人自身的iPSC后,修复病人iPSC基因组中的突变,分化为疾病相关细胞类型,然后移植给病人(如上图)。
▵ 项目概览
(Rui Wei et al., JHEP Reports, 2021 )
概念验证:基因修复后的iPSC来源肝细胞治疗肝豆状核变性
病因
Disease mechanism
图1 Copper Metabolism and ATP7B
(Aftab Ala et al., The lancet, 2007)
诊断和治疗
Diagnosis and Treatment
▵ 表1 现有治疗WD的药物5
▵ 表2 治疗WD的潜在疗法5
基于iPSC的创新疗法
Cell Therapy
图3. 利用CRISPR/Cas9和ssODN修复R778L突变
图4. Sanger测序确认R778L被修复(杂合)
全文链接6:
// 参考文献 //
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5. Jiayin Yang, PhD thesis. HKU
6. Wei R, Yang J, Cheng CW, Ho WI, Li N, Hu Y, Hong X, Fu J, Yang B, Liu Y, Jiang L, Lai WH, Au KW, Tsang WL, Tse YL, Ng KM, Esteban MA, Tse HF, CRISPR-targeted genome editing of human induced pluripotent stem cell-derived hepatocytes for the treatment of Wilson’s disease, JHEP Reports (2021), doi: https://doi.org/10.1016/j.jhepr.2021.100389.
编辑:Jianquan Zhang