CRISPR in Action: Unlocking New Cures for Human Diseases
DOI:
https://doi.org/10.71222/jgstag25Keywords:
CRISPR -cas9, gene editing, sickle cell disease, car-t therapy, bioethics, off-target effectsAbstract
The groundbreaking discovery of CRISPR -Cas9 technology has fundamentally opened new avenues for modern medicine, enabling the highly precise editing of the human genome to effectively treat a wide array of genetic and chronic diseases. This comprehensive review paper first introduces the fascinating natural origins of the CRISPR -Cas system within bacterial adaptive immunity. It subsequently details its sophisticated mechanism as a revolutionary gene-editing tool, specifically examining the structural features and endonuclease activity of the Cas9 protein, the precise targeting function of single guide RNA (sgRNA), and the subsequent cellular DNA repair mechanisms, namely non-homologous end joining (NHEJ) and homology-directed repair (HDR). Furthermore, the research highlights key clinical applications that have recently transitioned from the laboratory to patient care. This ranges from Casgevy, the landmark first FDA-approved CRISPR therapy for sickle cell disease in 2023, to highly promising emerging strategies aimed at complete HIV eradication and the development of enhanced CAR-T cell therapies for refractory cancers. While CRISPR technology demonstrates significant long-term potential for cost-effectiveness by offering unprecedented "one-time cures," several critical challenges persist. Its current exorbitant clinical price, the persistent risk of unintended off-target genomic effects, and complex ethical boundaries—particularly concerning heritable germline editing—remain major hurdles to global implementation. This paper concludes that while CRISPR is undeniably poised to transform the landscape of genomic medicine, further scientific breakthroughs, robust regulatory frameworks, and substantial cost reduction strategies are absolutely essential to ensure its widespread clinical accessibility and safety.References
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