CRISPR-CAS9: discovery and future therapeutic

Abstract

Abstract
CRISPR_Cas9 technology has quickly evolved into one of the most powerful tools in molecular biology, enabling precise
and efficient genome editing with a wide ranging of biomedical applications. Its ability to selectively target and modify
specific DNA or RNA sequences has new avenues in the treatment of cancer, inherited disorders, neurodegenerative
diseases, and viral infections. In malignant study, CRISPR has been employed to disrupt oncogenic drivers like KRAS and
MYC, restore tumor suppressor pathways involving TP53 and BRCA1, and optimize immunotherapy strategies through
engineered CAR-T cells. Similarly, in genetic disorders like Duchenne muscular dystrophy and Leber congenital amaurosis,
CRISPR-mediated exon skipping and targeted repair approaches have demonstrated significant therapeutic potential. In the
field of neurodegenerative diseases, the system has shown promise in modulating genes implicated in Alzheimer disease,
particularly APP, BACE1, and APOE, offering novel insights into disease modification. Furthermore, CRISPR-based RNAtargeting
systems like Cas13 have been successfully applied in controlling viral pathogens, including HIV and SARS-CoV-
2, not only for therapeutic interventions but also for rapid and sensitive diagnostics through platforms like SHERLOCK and
SHINE. Overall, CRISPR represents a transformative advance in precision medicine. While challenges such as off-target
effects, efficiency of delivery, and ethical considerations remain, continuous progress in optimizing the technology supports
its future integration into clinical practice as a versatile therapeutic and diagnostic platform.