摘要
CRISPR and adeno-associated virus are becoming powerful tools to remedy genetic disorders in somatic cells of adulthood.A recent study published in Science(Matharu et al.,2019)safely targeted the non-coding genomic region of Sim1 and MC4R with rAAV packed with CRISPRa,and successfully rescued the obesity syndrome caused by hap?loinsufficiency in a murine model,which shed light on their potential therapeutic applications in the future(Matharu et al.,2019).
基金
The work was supported by grants from National Key Research and Development Program of China(Grant No.2017YFA0103902)
The National Natural Science Foundation of China(Grant Nos.81570760 and 31771283)
the Fundamental Research Funds for the Central Universities of Tongji University.
