In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
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DOI: 10.1038/s41586-021-03534-y
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- Lisa N. Kasiewicz & Souvik Biswas & Aaron Beach & Huilan Ren & Chaitali Dutta & Anne Marie Mazzola & Ellen Rohde & Alexandra Chadwick & Christopher Cheng & Sara P. Garcia & Sowmya Iyer & Yuri Matsumot, 2023. "GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy," Nature Communications, Nature, vol. 14(1), pages 1-10, December.
- Elliot H. Choi & Susie Suh & Andrzej T. Foik & Henri Leinonen & Gregory A. Newby & Xin D. Gao & Samagya Banskota & Thanh Hoang & Samuel W. Du & Zhiqian Dong & Aditya Raguram & Sajeev Kohli & Seth Blac, 2022. "In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration," Nature Communications, Nature, vol. 13(1), pages 1-14, December.
- Hongzhi Zeng & Qichen Yuan & Fei Peng & Dacheng Ma & Ananya Lingineni & Kelly Chee & Peretz Gilberd & Emmanuel C. Osikpa & Zheng Sun & Xue Gao, 2023. "A split and inducible adenine base editor for precise in vivo base editing," Nature Communications, Nature, vol. 14(1), pages 1-14, December.
- Zhenxing Yu & Zhike Lu & Jingjing Li & Yingying Wang & Panfeng Wu & Yini Li & Yangfan Zhou & Bailun Li & Heng Zhang & Yingzheng Liu & Lijia Ma, 2022. "PEAC-seq adopts Prime Editor to detect CRISPR off-target and DNA translocation," Nature Communications, Nature, vol. 13(1), pages 1-13, December.
- Guiquan Zhang & Yao Liu & Shisheng Huang & Shiyuan Qu & Daolin Cheng & Yuan Yao & Quanjiang Ji & Xiaolong Wang & Xingxu Huang & Jianghuai Liu, 2022. "Enhancement of prime editing via xrRNA motif-joined pegRNA," Nature Communications, Nature, vol. 13(1), pages 1-12, December.
- Qinchang Chen & Guohui Chuai & Haihang Zhang & Jin Tang & Liwen Duan & Huan Guan & Wenhui Li & Wannian Li & Jiaying Wen & Erwei Zuo & Qing Zhang & Qi Liu, 2023. "Genome-wide CRISPR off-target prediction and optimization using RNA-DNA interaction fingerprints," Nature Communications, Nature, vol. 14(1), pages 1-17, December.
- Yue Xu & Shihao Ma & Haotian Cui & Jingan Chen & Shufen Xu & Fanglin Gong & Alex Golubovic & Muye Zhou & Kevin Chang Wang & Andrew Varley & Rick Xing Ze Lu & Bo Wang & Bowen Li, 2024. "AGILE platform: a deep learning powered approach to accelerate LNP development for mRNA delivery," Nature Communications, Nature, vol. 15(1), pages 1-16, December.
- Dominique L. Brooks & Manuel J. Carrasco & Ping Qu & William H. Peranteau & Rebecca C. Ahrens-Nicklas & Kiran Musunuru & Mohamad-Gabriel Alameh & Xiao Wang, 2023. "Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing," Nature Communications, Nature, vol. 14(1), pages 1-9, December.
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