Abstract
CRISPR-Cas9 gene editing has emerged as a transformative tool in pharmaceuticals, revolutionizing the understanding and treatment of genetic diseases. This review explores the current landscape and future potential of CRISPRCas9 technology in the pharmaceutical domain. Examining its fundamentals, including the CRISPR-Cas9 system and mechanisms, reveals unparalleled precision in disease modeling, drug discovery and targeted therapies. Challenges like offtarget effects and regulatory complexities are addressed alongside advancements in CRISPR technology beyond Cas9, offering enhanced precision and expanded therapeutic horizons. The significance of CRISPR-Cas9 in shaping the future of pharmaceuticals is evident in its potential applications for rare diseases, complex disorders and its integration with other pharmaceutical approaches, promising groundbreaking therapies. Ethical considerations and regulatory frameworks necessitate responsible innovation while predicting a profound impact on personalized medicine. The future of CRISPR-Cas9 in pharmaceuticals lies in refining delivery methods, addressing ethical concerns and exploring synergies with emerging technologies. This article underscores the ongoing evolution and immense potential of CRISPR-Cas9 in revolutionizing healthcare and therapeutics.
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