TITLE: CRISPR-CASAS THERAPEUTIC TOOL: TRANSLATIONAL PERSPECTIVES FROM PRECLINICAL STUDIES TO CLINICAL TRIALS.

Rutuja Vilas Sahajrao , Sanchit Ramrao Magar , Snehal Anil Bhosle , Tarnnum Sameer Shaikh

1)CRISPR-Cas9 genome editing 2)Therapeutic gene editing strategies 3)Clinical genome editing applications 4)Base and prime editing 5)Epigenome and transcriptome editing 6)Gene therapy delivery systems 7)Off-target editing effects 8)Sickle cell disease therapy 9)?-thalassemia gene therapy 10)Cancer immunotherapy using CRISPR 11)Rare genetic disease treatment 12)Personalised precision medicine approaches

Abstract: Introduction- The CRISPR-Cas9 system has revolutionised genome editing by enabling efficient, programmable, and cost-effective modifications of DNA across diverse organisms. Since its adaptation from bacterial adaptive immunity, CRISPR-Cas9 has rapidly transitioned from preclinical models to early human clinical trials, establishing itself as a transformative platform in modern therapeutics. In vitro and in vivo studies have demonstrated its potential to correct disease-causing mutations, model complex pathologies, and accelerate drug discovery. Methodology- Clinical applications are already underway in blood disorders such as sickle cell disease and ?-thalassemia, where the FDA-approved therapy exagamglogene autotemcel (CASGEVY(TM)) represents the first CRISPR-based treatment. Additional trials, including EDIT-101 for Leber congenital amaurosis and NTLA-2001 for transthyretin amyloidosis, underscore its versatility across genetic and metabolic diseases. CRISPR is also being explored in oncology, infectious diseases, and rare disorders, while next-generation systems such as Cas12, Cas13, base and prime editors, and epigenome regulators are broadening its capabilities. Despite remarkable progress, challenges remain regarding off-target effects, delivery efficiency, immune responses, long-term safety, and ethical concerns, particularly in the context of germline editing. Conclusion -Advances in delivery platforms, high-fidelity nucleases, and computational tools are addressing these barriers, while integration with AI, nanotechnology, and synthetic biology promise to expand the precision and applicability of genome editing. In short, CRISPR-Cas9 is at the vanguard of genetic medicine as a tool for both study and treatment. Its clinical translation offers the unprecedented potential for one-time, curative therapies, but realising this promise will require not only technical refinement but also equitable access, global governance, and ethical responsibility.

Volume 13 Issue 10, October-2025

Pages : d231-d246