Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption.
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:
主题词
动物(Animals);小鼠(Mice);高草酸尿症, 原发性(Hyperoxaluria, Primary);醇氧化还原酶类(Alcohol Oxidoreductases);疾病模型, 动物(Disease Models, Animal);人类(Humans);纳米粒子(Nanoparticles);草酸盐类(Oxalates)
DOI
10.1016/j.ymthe.2024.10.003
PMID
39385468
发布时间
2025-01-10
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Molecular therapy
104-118页
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