CRISPR/Cas-system as a molecular tool for therapy of genetic diseases of animals and human
Prokaryote-specific CRISPR/Cas-system seems to function as molecular adaptive immune response against viruses and foreign DNA. Ability of the system to specifically recognize and hydrolyse genome locus by using specific short RNA is utilised in the modern technology called genome editing. In this review we shortly describe structure and function of the CRISPR/Cas system belonging to II class, its application in treatment of genetic diseases in mammals and human, and its disadvantages that limit its application in genetic therapy.