CRISPR - CAS MEDIATED GENOME EDITING FOR HUMAN DISEASE THERAPY: EMERGING APPLICATIONS, ADVANCEMENTS AND FUTURE PERSPECTIVES
Abstract
CRISPR - Cas - mediated genome editing has emerged as a transformative technology with considerable potential for the treatment of human diseases. The ability of CRISPR-Cas systems to recognize specific nucleic acid recognize specific nucleic acid sequences and modify targeted genetic regions has expanded the possibilities of precision medicine and gene therapy. This review provides an overview of the recent advancements in CRISPR - Cas - mediated genome editing and its emerging therapeutic applications in human diseases. Particular attention is given to conventional Cas 9 - based editing, and other programmable editing strategies that can enable more precise genetic modifications. The therapeutic potential of CRISPR - Cas technology is being investigated across a broad range of conditions, including inherited genetic disorders, hematological diseases, cancer, infectious diseases, metabolic disorders, and other complex diseases. The review also discusses important development in ex vivo and in vivo genome - editing approaches and the role of delivery systems in including therapeutic efficiency and tissues and specificity. Despite rapid progress, factors such as unintended genetic alterations, limited delivery efficiency, immune responses, editing variability, long term safety, and ethical considerations remain important areas for continued investigations. Future developments are expected to focus on improving editing precision, developing safer and more effective delivery platforms, expanding clinical applications, establishing robust approaches for long-terms monitoring. Overall CRISPR - Cas - mediated genome editing represents a promising platform for advancing targeted and personalized therapies for human disease.
