CRISPR is a revolutionary gene-editing technology that allows scientists to easily and precisely alter DNA sequences and modify gene function. Derived from a natural defense mechanism found in bacteria, the system consists of a guide RNA molecule and a Cas enzyme (most commonly Cas9). The guide RNA acts like a GPS, locating a specific target sequence of DNA, while the Cas enzyme acts like a pair of molecular scissors, making a precise cut at that location.
This ability to “cut and paste” DNA has immense implications across life sciences. In drug discovery, CRISPR is used to rapidly create in vitro (cell culture) and in vivo (animal) models of human disease, accelerating target validation. Clinically, it forms the basis of Gene Therapy and is being explored to correct disease-causing mutations directly in a patient’s cells to treat genetic disorders, infectious diseases, and various forms of cancer. While its precision makes it a powerful therapeutic tool, the technology continues to evolve, with ongoing research focused on improving delivery methods and ensuring long-term safety and efficacy.
