Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption.
通过 LNP-CRISPR-Cas9 介导的乙醇酸氧化酶破坏，高效安全地在体内治疗 1 型原发性高草酸尿症
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.10.003
Jiang Yanhong, Chen Shuanghong, Hsiao Shenlin, Zhang Haokun, Xie Da, Wang Zi Jun, Ren Wendan, Liu Mingyao, Liao Jiaoyang, Wu Yuxuan
