其他
Cell重磅:中科院杨辉组通过“剪刀手”将胶质细胞向神经元转分化,可治疗视力损伤和PD
图注:(上)CasRx通过靶向的降解Ptbp1 mRNA从而实现Ptbp1基因表达的下调。(中)视网膜下注射AAV-GFAP-CasRx-Ptbp1可以特异性的将视网膜穆勒胶质细胞转分化为视神经节细胞,转分化而来视神经节细胞可以和正确的脑区建立功能性的联系,并且提高永久性视力损伤模型小鼠的视力。(下)在纹状体中注射AAV-GFAP-CasRx-Ptbp1可以特异性的将星形胶质细胞转分化为多巴胺神经元,从而基本消除了帕金森疾病模型小鼠的运动症状。
代表性论文 |
• Zhou, C.,Sun, Y., Yan, R., Liu, Y., Zuo, E., Gu, C., Han, L., Wei Y., Zeng, R., Li, Y.(*), Zhou H.(*), Guo F.(*), Yang H.* (2019) Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis Nature 517: 275-278 |
• Zhou, H., Liu, J., Zhou, C., Gao, N., Rao, Z., Li, H., Hu, X., Li, C., Yao, X., Shen, X., Sun, Y., Wei, Y., Liu, F., Ying, W., Zhang, J., Tang, C., Zhang, X., Xu, H., Shi, L., Cheng, L., Huang, P. *, Yang, H.* (2018) In vivo simultaneous transcriptional activation of multiple genes in the brain using CRISPR/dCas9-activator transgenic mice Nat. Neurosci. 21: 440-446 |
• Zuo, E., Sun, Y., Wu, W., Yuan, T., Ying, W., Sun, H., Yuan, L., Steinmetz, L.*, Li, Y.*, Yang, H.* (2019) Cytosine base editor generates substantial off-target single nucleotide variants in mouse embryos Science 364: 289-292 |
• Yao, X.*, Zhang, M., Wang, X., Ying, W., Hu, X., Dai, P., Meng, F., Shi, L., Sun, Y., Yao, N., Zhong, W., Li, Y., Wu, K., Li, W.*, Chen, Z.*, Yang, H.* (2018) Tild-CRISPR allows for efficient and precise gene knockin in mouse and human cells Dev. Cell 45: 526-536 |
• Zuo, E.W., Huo, X.N., Yao, X., Hu, X.D., Sun, Y.D., Yin, J.H., He, B.B., Wang, X., Shi, L.Y., Ping, J., Wei, Y., Ying, W.Q., Wei, W., Liu, W.J., Tang, C., Li, Y.X., Hu, J.Z.* and Yang, H.* (2017) CRISPR/Cas9-mediated targeted chromosome elimination Genome Biol. 18: 224 |
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