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

作者：Yanhong Jiang, Shuanghong Chen, Shenlin Hsiao, Haokun Zhang, Da Xie, Zijun Wang, Wendan Ren, Mingyao Liu, Jiaoyang Liao, Yuxuan Wu · 发表于：Molecular Therapy · 年份：2024 · DOI：10.1016/j.ymthe.2024.10.003 · 被引用次数：20 · 研究领域：Kidney Stones and Urolithiasis Treatments、Porphyrin Metabolism and Disorders、Biomedical Research and Pathophysiology