GENOME EDITING: CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT (CRISPR/CAS9)

Abstract

The increase in the prevalence of oncological diseases and genetic diseases among the world population remains an open problematic issue of the scientific world, connected with the need to develop modern methods of treatment. The main goal of conducting research is to reduce the overall rate of mortality and disability among the world population. One of the promising technologies of genetic engineering is the Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR/Cas9) technology, which allows targeted editing of the genome. The use of this technique allows to solve the treatment of such pathologies as: oncological diseases, sickle cell anemia, β-thalassemia, amyloidosis, nanotechnology, Duchenne muscular dystrophy, Parkinson's disease caused by genetic causes, digenic and monogenic hearing loss, etc. At this stage of the development of this technology, it is not available for the treatment of genetic and oncological diseases in general medicine. Clinical trials, development of application strategies and prevention of side effects in treatment using molecular scissors are being conducted. The question of the safe use of CRISPR/Cas9 is related to the possibility of unwanted mutations in the genetic material, with the lack of full prediction of the consequences. Choosing the optimal way to deliver CRISPR/Cas9 to the target and developing and improving the technology to prevent the occurrence of unwanted indels are no less urgent issues. The prospects of using these technologies give hope for reducing the mortality and disability of patients with pathologies that are currently difficult to treat or are incurable. Taking into account the percentage of efficiency, the use of CRISPR/Cas9 technology, in contrast to ZFN (Zinc-Finger Nucleases) or TALEN (Transcription Activator-Like Effector Nucleases), is more optimal.