crispr-cas9

Increased TBX6 gene dosages induce congenital cervical vertebral malformations in humans and mice
Xiaojun Ren, Nan Yang, Nan Wu, Ximing Xu, Weisheng Chen, Ling Zhang, Yingping Li, Ren-Qian Du, Shuangshuang Dong, Sen Zhao, Shuxia Chen, Li-Ping Jiang, Lianlei Wang, Jianguo Zhang, Zhihong Wu, Li Jin, Guixing Qiu, James R Lupski, Jiangang Shi, Feng Zhang, Pengfei Liu

Journal of Medical Genetics Jun 2020, 57 (6) 371-379; DOI: 10.1136/jmedgenet-2019-106333
Mutation-specific Fabry disease patient-derived cell model to evaluate the amenability to chaperone therapy
Malte Lenders, Franciska Stappers, Christoph Niemietz, Boris Schmitz, Michel Boutin, Paula Johanna Ballmaier, Andree Zibert, Hartmut Schmidt, Stefan-Martin Brand, Christiane Auray-Blais, Eva Brand

Journal of Medical Genetics Aug 2019, 56 (8) 548-556; DOI: 10.1136/jmedgenet-2019-106005
Applications and advances of CRISPR-Cas9 in cancer immunotherapy
An-Liang Xia, Qi-Feng He, Jin-Cheng Wang, Jing Zhu, Ye-Qin Sha, Beicheng Sun, Xiao-Jie Lu

Journal of Medical Genetics Jan 2019, 56 (1) 4-9; DOI: 10.1136/jmedgenet-2018-105422

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