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Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
Adam C. Wilkinson, Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth, Chika Morita, Hiromitsu Nakauchi, Matthew H. Porteus
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Article Pages: 1 Open access contents
In-situ generation of large numbers of genetic combinations for metabolic reprogramming via CRISPR-guided base editing
Yu Wang, Haijiao Cheng, Yang Liu, Ye Liu, Xiao Wen, Kun Zhang, Xiaomeng Ni, Ning Gao, Liwen Fan, Zhihui Zhang, Jiao Liu, Jiuzhou Chen, Lixian Wang, Yanmei Guo, Ping Zheng, Meng Wang, Jibin Sun, Yanhe Ma
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Article Pages: 1 Open access contents
TALEN outperforms Cas9 in editing heterochromatin target sites
Surbhi Jain, Saurabh Shukla, Che Yang, Meng Zhang, Zia Fatma, Manasi Lingamaneni, Shireen Abesteh, Stephan Thomas Lane, Xiong Xiong, Yuchuan Wang, Charles M. Schroeder, Paul R. Selvin, Huimin Zhao
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Article Pages: 1 Open access contents
CRISPR technologies and the search for the PAM-free nuclease
Daphne Collias, Chase L. Beisel
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Article Pages: 1 Open access contents
Engineered dual selection for directed evolution of SpCas9 PAM specificity
Gregory W. Goldberg, Jeffrey M. Spencer, David O. Giganti, Brendan R. Camellato, Neta Agmon, David M. Ichikawa, Jef D. Boeke, Marcus B. Noyes
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Article Pages: 1 Open access contents
SLE non-coding genetic risk variant determines the epigenetic dysfunction of an immune cell specific enhancer that controls disease-critical microRNA expression
Guojun Hou, Isaac T. W. Harley, Xiaoming Lu, Tian Zhou, Ning Xu, Chao Yao, Yuting Qin, Ye Ouyang, Jianyang Ma, Xinyi Zhu, Xiang Yu, Hong Xu, Dai Dai, Huihua Ding, Zhihua Yin, Zhizhong Ye, Jun Deng, Mi Zhou, Yuanjia Tang, Bahram Namjou, Ya Guo, Matthew T. Weirauch, Leah C. Kottyan, John B. Harley, Nan Shen
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Article Pages: 1 Open access contents