Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are associated with lowered blood lipid levels, making Angptl3 an attractive therapeutic target for the treatment of human lipoprotein metabolism disorders. In this study, we developed a lipid nanoparticle delivery platform carrying Cas9 me...

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Библиографические подробности
Опубликовано в: :Proc Natl Acad Sci U S A
Главные авторы: Qiu, Min, Glass, Zachary, Chen, Jinjin, Haas, Mary, Jin, Xin, Zhao, Xuewei, Rui, Xuehui, Ye, Zhongfeng, Li, Yamin, Zhang, Feng, Xu, Qiaobing
Формат: Artigo
Язык:Inglês
Опубликовано: National Academy of Sciences 2021
Предметы:
Biological Sciences
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC7958351/
https://ncbi.nlm.nih.gov/pubmed/33649229
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.2020401118
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