Abstract

AN OVERVIEW OF THE CRISPR SYSTEM AND ITS DEVELOPMENT IN THE FIELD OF PHARMACEUTICAL INDUSTRY

*Venkatakrishnan R., Daphne Sherine, Kabilan S. and Thirupathi S.

Abstract

A naturally occurring genome editing system that bacteria use as an immune defence is the basis for CRISPR-Cas9. CRISPR-based gene editing is a powerful tool that enables mammalian genome engineering. Chemically modified or DNA-substituted nucleotides are also well tolerated at particular positions and up to 70% of the crRNA DNA specificity area. Gene-editing activity is also maintained by changes to the phosphorothioate backbone in the crRNA DNA Specificity region. The most sophisticated and extensively used CRISPR-Cas system, CRISPR-Cas9, has been improved by the proofof- principle studies by giving researchers a better understanding of genes’ role in medicine absorption and disposal. The prevention and treatment of human diseases is a major area of focus for CRISPR-based gene editing. Genome editing is currently employed in research facilities to study diseases in cells and animal models. Genome editing using CRISPR techniques in CRISPR-Cas9 gene modification is very likely useful for treating incurable mammalian genetic disorders but can be treated at the embryonic development stage. This is because CRISPR tools are now being developed that can be used to study cancer biology as well as neurodegenerative disease, blood disorders, cancer, and other diseases. But when human genomes are altered by genome editing, utilizing tools like CRISPR-Cas9, ethical concerns are raised. The majority of the modifications brought about by genome editing are only seen in somatic cells, which are cells other than egg and sperm cells (germline cells).

Keywords: CRISPR, Gene-editing, CRISPR-Cas9, DNA Modification, Mammalian diseases.