Good evening! Does anybody know, what CRISPR gene editing is? I've read that this method is considered as a promising direction in the treatment of hereditary diseases, and recently (in 2020) it's research in vivo was started. But, to be honest, I don't know at all what it is. Could anybody explain?
Ok, CRISPR gene editing is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified. You want to know exact procedure?
Long story short: 1) Decide which gene you want to cut. 2) Design a gRNA to target a specific PAM sequence near that region. 3) Express that gRNA in the cell of interest in addition to an endonuclease protein such as Cas9 or Cpf1. 4) Voila! The DNA will be cut at that position and some of the resulting cells will have loss-of-function mutations in the target gene.