摘要
The steady progress in genome editing, especially genome editing based on the use of clustered regularly interspaced short palindromic repeats(CRISPR) and programmable nucleases to make precise modifications to genetic material, has provided enormous opportunities to advance biomedical research and promote human health. The application of these technologies in basic biomedical research has yielded significant advances in identifying and studying key molecular targets relevant to human diseases and their treatment. The clinical translation of genome editing techniques offers unprecedented biomedical engineering capabilities in the diagnosis, prevention, and treatment of disease or disability. Here, we provide a general summary of emerging biomedical applications of genome editing, including open challenges. We also summarize the tools of genome editing and the insights derived from their applications, hoping to accelerate new discoveries and therapies in biomedicine.
基金
supported by the National Natural Science Foundation of China (81830004, 31922046, 31770057, 31722036, 31930016, 31870893)
the Sanming Project of Medicine in Shenzhen (SZSM202011017)
the National Key Research and Development Program of China (2018YFA0801401, 2019YFA0110800, 2018YFA0107703, 2019YFA0110000, 2020YFA0707800, 2020YFA0707600)
the Strategic Priority Research Program of the Chinese Academy of Sciences (XDA16030403, XDA16010503)
Beijing Municipal Science & Technology Commission (Z181100001318009)
Beijing Advanced Innovation Center for Genomics at Peking University
the Peking-Tsinghua Center for Life Sciences
the National Major Science & Technology Project for Control and Prevention of Major Infectious Diseases in China (2018ZX10301401)
