Adeno-Associated Virus-Mediated RNAi against Mutant Alleles Attenuates Abnormal Calvarial Phenotypes in an Apert Syndrome Mouse Model

Molecular Therapy - Nucleic Acids - Tập 13 - Trang 291-302 - 2018
Fengtao Luo1, Yangli Xie1, Zuqiang Wang1, Junlan Huang1, Qiaoyan Tan1, Xianding Sun1, Fangfang Li1, Can Li1, Mi Liu1, Dali Zhang1, Meng Xu1, Nan Su1, Zhenhong Ni1, Wanling Jiang1, Jinhong Chang1, Hangang Chen1, Shuai Chen1, Xiaoling Xu2, Chuxia Deng2, Zhugang Wang3
1Laboratory for the Rehabilitation of Traumatic Injuries, Center of Bone Metabolism and Repair, State Key Laboratory of Trauma, Burns and Combined Injury, Trauma Center, Research Institute of Surgery, Daping Hospital, Third Military Medical University, Chongqing 400042, China
2Faculty of Health Sciences, University of Macau, Macau SAR, China
3State Key Laboratory of Medical Genomics, Research Center for Experimental Medicine, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China

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Molecular Therapy - Nucleic Acids

Tập 13

291-302

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