Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption.

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第一作者： Yanhong,Jiang

第一单位： Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai 200241, China; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China.

作者： Yanhong,Jiang ^[1] ; Shuanghong,Chen ^[1] ; Shenlin,Hsiao ^[1] ; Haokun,Zhang ^[2] ; Da,Xie ^[2] ; Zi Jun,Wang ^[2] ; Wendan,Ren ^[2] ; Mingyao,Liu ^[3] ; Jiaoyang,Liao ^[4] ; Yuxuan,Wu ^[5]

作者单位： Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai 200241, China; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China. ^[1] YolTech Therapeutics, Shanghai 201109, China. ^[2] Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai 200241, China; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China. Electronic address: myliu@bio.ecnu.edu.cn. ^[3] Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai 200241, China; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China. Electronic address: jyliao@bio.ecnu.edu.cn. ^[4] Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai 200241, China; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China; YolTech Therapeutics, Shanghai 201109, China. Electronic address: yxwu@bio.ecnu.edu.cn. ^[5]

关键词 CRISPR/Cas9 LNPs PH1 PH1 treatment genome editing glycolate oxidase lipid nanoparticles primary hyperoxaluria type 1 urine oxalate