CRISPR/Cas9 therapeutics: progress and prospects

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from…

• NN
  National Natural Science Foundation of China

  Award: 81870331
• NS
  Natural Science Foundation of Shandong Province

  Award: ZR2021MH280
• QM
  Qingdao Municipal Science and Technology Bureau

  Award: No.21-1-4-rkjk-12-nsh