CRISPR/Cas9 system: A versatile gene editing tool in disease prevention and treatment

Abstract

With the development of genome editing as a promising mechanism for disease prevention and treatment, clustered regularly interspaced short palindromic repeats (CRISPR) is a robust and multiplexed tool that is able to address the challenges faced by its predecessors such as ZincFinger Nucleases (ZFNs) and Transcription Activator-like Effector Nuclease (TALENs). The findings indicate that CRISPR-Cas9 is more precise, convenient and cost effective in comparison with its forerunners in terms of DNA cleavage. Recent advancements of this tool have enabled researchers and scientists to adopt treatment options for diseases like cancer, AIDS, muscular dystrophy, blood disorders, cystic fibrosis, Huntington’s disease etc. Despite massive potential of this tool to detect and modify genes responsible for various diseases, human trials remain the most controversial factor in scientific community and academia. Hence, more research is needed to explore more possibilities and fill the gaps in information that can prove to be crucial for achieving desired outcomes in disease prevention and treatment.