A genome editing technology and capabilities of its application in cellular neurobiology

Abstract

A number of fundamental breakthroughs in cellular and molecularbiology provided the basis for several modern sophisticatedapproaches to modeling of human neurological (primarily neurodegenerative)diseases. In particular, targeted genome editingby artificial nuclease systems (CRISPR/CAS9, etc.) enables ahighly specific correction of genetic defects at the cellular level.An especially promising area is application of the genome editingtechnology in specialized neurons and induced pluripotent stemcells (iPSCs) derived from fibroblasts of patients with inheritedforms of neurodegeneration by cell reprogramming. The articleprovides a brief analysis of programmable nuclease systems anddescribes mechanisms of their activity as well as advantages, disadvantages,and capabilities of their applications in modelingand correction of neurodegenerative diseases. The authors generalizetheir own experience in cellular modeling of the PARK2type of Parkinsons disease on the culture of dopaminergic neuronsdifferentiated from iPSCs. The article provides preliminarydata related to the capability of editing the cellular genome atmutant sites PARK2.

Keywords

• pluripotent stem cells
• genome editing, crispr/cas9
• experimental neurobiology