Abstract

CRISPR/CAS9: HISTORY AND COMPONENTS, MECHANISM, APPLICATION

*Sowbarnika S.

ABSTRACT

CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) is a precise tool in gene editing that has revolutionized in life science, medicine and drug delivery. It was discovered in the 18th century as an astonishing sequence in bacteria and archaea, which developed adaptive immunity through the incorporation of spacers from foreign nucleic acids to combat invading viruses. Two women, Emmanuelle Charpentier and Jennifer Doudna, who significantly advanced the field of genetic manipulation, were awarded the Noble Prize in 2020 for their biochemical studies on CRISPR/Cas9. The basic mechanism of CRISPR involves recognition, cleavage, and repair of DNA. This process is achieved through insertion, deletion, or single base changes. CRISPR’s evolutionary advancements in molecular biology have increased its therapeutic potential due to its high efficiency and effectiveness, earning it name ―Genetic Scissors‖. It plays a crucial role in gene therapy, gene editing and the treatment togenetic diseases. CRISPR has tremendous applications in medicine, including treating cystic fibrosis, sickle cell anemia, cancer and Duchenne Muscular Dystrophy. Although CRISPR has shown immense potential across various sectors, many applications remain disapproved due to challenges like immunogenicity of Cas9 protein, off- target effects and the risk of cancer. The review discussed the History, mechanism of action and therapeutic uses of CRISPR/Cas9.

Keywords: CRISPR/Cas9, sgRNA, Genetic Scissors, off- target effects.