个人简介

刘雄昊，博士，研究员，博士生导师。现任湖南省遗传学会理事，湖南省生物医学工程学会理事，湖南省医学会医学遗传学专业委员会常务委员，湖南省免疫学会免疫治疗分会委员。承担国家重点研发计划课题、863项目课题、973项目课题、国家自然科学基金项目10余项。以第一作者或通讯作者在Signal Transduction and Targeted Therapy，Cancer Res，Hum Gene Ther等学术期刊发表论文20余篇，申请发明专利4项，已获授权发明专利2项。

研究方向

1）利用基因编辑和干细胞技术开展重大疾病的细胞与基因治疗。建立iPS细胞系，利用基因编辑技术对其进行工程化改造，将经基因修饰的iPSC细胞定向分化而来的特定类型细胞开展重大疾病的治疗。

2）肿瘤的基因治疗。开发基于干细胞外泌体的递送系统，装载抗癌基因或免疫调节因子，实现靶向肿瘤治疗研究。

3）肌萎缩侧索硬化症（ALS）的发病机制与治疗研究。肌萎缩侧索硬化症（ALS，俗称"渐冻症"）是一种致命的神经退行性疾病，其特征是大脑和脊髓运动神经元进行性退化，导致肌肉萎缩、瘫痪，最终因呼吸衰竭死亡。约10%病例为家族遗传性，利用患者来源iPSC或基因编辑技术，建立携带ALS致病基因突变的iPSC源性运动神经元（iPSC-MN）模型，通过多组学分析（转录组、蛋白组、代谢组）研究运动神经元退化的关键分子通路；建立高通量药物筛选平台，鉴定具有神经保护作用的小分子化合物；探索干细胞移植治疗ALS的潜力。

近年代表性论文：

1.Zhao, M., Wang, J., Liu, M., Xu, Y., Huang, J., Zhang, Y., He, J., Gu, A., Liu, M*., and Liu, X*. (2024). KIF1A, R1457Q, and P1688L Mutations Induce Protein Abnormal Aggregation and Autophagy Impairment in iPSC-Derived Motor Neurons. Biomedicines, 12(8), 1693.

2.Liu, M., Zhang, Y., He, J., Liu, W., Li, Z., Zhang, Y., Gu, A., Zhao, M., Liu, M*., and Liu, X*. (2024). Fusion with ARRDC1 or CD63: A Strategy to Enhance p53 Loading into Extracellular Vesicles for Tumor Suppression. Biomolecules, 14(5), 591.

3.Liu, D., Wei, B., Liang, L., Sheng, Y., Sun, S., Sun, X., Li, M., Li, H., Yang, C., Peng, Y., Xie, Y., Wen, C., Chen, L., Liu, X*., Chen, X*., Liu, H*., and Liu, J*. (2024). The Circadian Clock Component RORA Increases Immunosurveillance in Melanoma by Inhibiting PD-L1 Expression. Cancer research, 84(14), 2265.

4.Gu, A., Zhang, Y., He, J., Zhao, M., Ding, L., Liu, W., Xiao, J., Huang, J., Liu, M*., and Liu, X*. (2024). Chronic Oxidative Stress and Stress Granule Formation in UBQLN2 ALS Neurons: Insights into Neuronal Degeneration and Potential Therapeutic Targets. International journal of molecular sciences, 25(24), 13448.

5.Zhang, Y., Zeng, B., Gu, A., Kang, Q., Zhao, M., Peng, G., Zhou, M., Liu, W., Liu, M., Ding, L., Liang, D., Liu, X*, and Liu, M*. (2022). Damaged DNA Is an Early Event of Neurodegeneration in Induced Pluripotent Stem Cell-Derived Motoneurons with UBQLN2P497H Mutation. International journal of molecular sciences, 23(19), 11333.

6.Peng, G., Gu, A., Niu, H., Chen, L., Chen, Y., Zhou, M., Zhang, Y., Liu, J., Cai, L., Liang, D., Liu, X*, and Liu, M*. (2022). Amyotrophic lateral sclerosis (ALS) linked mutation in Ubiquilin 2 affects stress granule assembly via TIA-1. CNS neuroscience & therapeutics, 28(1), 105.

7.Zeng, B., Zhou, M., Liu, B., Shen, F., Xiao, R., Su, J., Hu, Z., Zhang, Y., Gu, A., Wu, L., Liu, X*, and Liang, D*. (2021). Targeted addition of mini-dystrophin into rDNA locus of Duchenne muscular dystrophy patient-derived iPSCs. Biochemical and biophysical research communications, 545, 40.

8.Hu, Q., Zhang, Y., Wang, P., Zhou, M., Hu, Z., Liu, C., Liu, M., Wu, L., Liu, X*, and Liang, D*. (2021). IL-24 armored CAR19-T cells show enhanced antitumor activity and persistence. Signal transduction and targeted therapy, 6(1), 14.

9.Wu, Z., Liu, W., Wang, Z., Zeng, B., Peng, G., Niu, H., Chen, L., Liu, C., Hu, Q., Zhang, Y., Pan, M., Wu, L., Liu, M*., Liu, X*., and Liang, D. (2020). Mesenchymal stem cells derived from iPSCs expressing interleukin-24 inhibit the growth of melanoma in the tumor-bearing mouse model. Cancer cell international, 20, 33.

10.Liu, X., Liu, M., Wu, L., & Liang, D. (2018). Gene Therapy for Hemophilia and Duchenne Muscular Dystrophy in China. Human gene therapy, 29(2), 146.

11.Liu, B., Chen, F., Wu, Y., Wang, X., Feng, M., Li, Z., Zhou, M., Wang, Y., Wu, L., Liu, X*., and Liang, D*. (2017). Enhanced tumor growth inhibition by mesenchymal stem cells derived from iPSCs with targeted integration of interleukin24 into rDNA loci. Oncotarget, 8(25), 40791.

12.Liu, X., Wu, Y., Li, Z., Yang, J., Xue, J., Hu, Y., Feng, M., Niu, W., Yang, Q., Lei, M., Xia, J., Wu, L., and Liang, D*. (2012). Targeting of the human coagulation factor IX gene at rDNA locus of human embryonic stem cells. PloS one, 7(5), e37071.

13.Liu, X., Liu, M., Xue, Z., Pan, Q., Wu, L., Long, Z., Xia, K., Liang, D*., and Xia, J*. (2007). Non-viral ex vivo transduction of human hepatocyte cells to express factor VIII using a human ribosomal DNA-targeting vector. Journal of thrombosis and haemostasis , 5(2), 347.