Abstract

CRISPER VS RNAI: COMPARATIVE REVIEW IN GENE SILENCING APPLICATION

*Rupam Kumari, *Sana Nusrat Praween, *Sachin Kumar

ABSTRACT

Gene silencing is the regulation of gene expression in a cell to prevent the expression of a certain gene. Gene silencing can occur during either transcription or translation and is often used in research. Gene silencing is a relatively new treatment technique that makes use of the body‘s natural processes to control disease by suppressing or ‗silencing‘ specific genes that are associated with certain diseases.in this context, silencing means temporarily blocking a specific gene‘s message that would otherwise trigger an unwanted effect. RNA-Interference is a biological process in which RNA molecules are involved in sequence-specific by double –stranded RNA .Deficiencies in RNAI pathway can lead to various issues, including impaired immune responses and developmental abnormalities. Treatment approaches focus on restoring or supplementing the RNAi pathways, often using RNAi based therapeutics to target specific genes or viralinfection. CRISPER-Cas9 is a gene- editing technology causing a major upheaval in biomedical research. Other potential clinical applications include gene therapy, treating infections diseases such as OCA2 gene and engineering autologous patient material to treat cancer and other disease. In this review we will give an overview of CRISPER-Cas9 with an emphasis on how it may impact on the specialty of paediatrics .CRISPER –Cas9 is its potential use to treat genetic disorders caused by single gene mutations. Examples of such diseae include cystic fibrosis(CF), Duchenne‘s muscular dystrophy(DMD) and haemoglobinopathiies.

Keywords: CRISPER –Cas9, RNAi, Gene silencing, OCA2 gene, Transcription, DMD.