摘要
The continuous development of various gene therapies has brought new light to the treatment of genetic diseases.Among them,therapies targeting monogenic diseases are relatively progressive and promising due to the explicit and clear pathogenesis.The discovery and development of clustered regularly interspaced short palindromic repeats/associated nuclease(CRISPR/Cas)and related technologies was undoubtedly an extraordinary leap forward for gene therapy.
基金
supported by grants from the Ministry of Science and Technology of China(2018YFA0107900,92168103,32171417,82001140,2019CXJQ01)
the National Nature Science Foundation,and Shanghai Municipal Government,Peak Disciplines(TypeⅣ)of Institutions of Higher Leaning in Shanghai。
