Review Article The Emerging Role Of Crispr In The Treatment Of Rare Genetic Disorders

Abstract

Although each individual rare genetic disorder is uncommon, collectively they represent a significant global health challenge. The clustered regularly interspaced short palindromic repeats (CRISPR) genome-editing technology has attracted substantial attention in medicine due to its adaptability, simple design, high efficiency, and precision, driving rapid progress in the field. It supports scientific research by facilitating disease modelling, enhancing crop strains, and creating treatments for genetic conditions. In the medical field, it has been used or studied for disorders such as sickle cell disease and other inherited conditions. Unlike earlier gene-editing methods, it offers greater speed, lower costs, and higher precision. The technology functions by identifying specific DNA sequences using specialized proteins, allowing not just gene editing but also the control of gene expression.