Abstract
The evolution of technology allowed the discovery of the genetic alterations responsible for various genetic diseases. The treatment options generally available for these pathologies are mainly focused on treating the symptoms, but not to restore the altered genetic sequences responsible for the disease phenotype. Clustered regularly interspaced short palindromic repeats-CRISPR associated (CRISPR/Cas) systems can be used for gene therapy and the fundamental principle is based on genome editing and regulation of physiological phenomena. CRISPR/Cas system was discovered as a component of the bacterial immune system and was repurposed as a genome editing tool that is now broadly used in molecular biology applications. This chapter focuses on the discovery of the CRISPR/Cas systems in bacteria, the description of the components and features of CRISPR/Cas system types, and on describing the molecular mechanisms used for genome editing. Other aspects of this chapter include the applications of CRISPR/Cas9 for biomarker discovery, as an editing tool for gene therapy, and its use for the diagnosis of infectious diseases. Finally, the challenges of using CRISPR/Cas9 as a gene editing tool are depicted and explained.
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