Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption
Yanhong Jiang, Shuanghong Chen, Shenlin Hsiao, Haokun Zhang, Da Xie, Zijun Wang, Wendan Ren, Mingyao Liu, Jiaoyang Liao, Yuxuan Wu
Topics & Concepts
CRISPRIn vivoPrimary hyperoxaluriaChemistryOxidase testBiologyBiochemistryEnzymeGeneticsGeneKidneyKidney Stones and Urolithiasis TreatmentsPorphyrin Metabolism and DisordersBiomedical Research and Pathophysiology