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Vol. 01 No. 03 (March-2026) : Vol. 01, No. 03-March.2026-BTS INTERNATIONAL ADVANCED PHARMACEUTICAL SCIENCES JOURNAL

ENGINEERING THE FUTURE: CRISPR-BASED GENE EDITING IN PERSONALIZED THERAPEUTICS

Abstract

Genome editing technologies (GED) have revolutionized biomedical research and therapeutic development, allowing precise modifications of DNA in living cells. CRISPR-Cas9 is unique among GED tools because of its effectiveness, ease of use, and versatility. Although CRISPR-Cas9 was initially discovered to be an immune defense mechanism in bacteria, it has since evolved into a powerful gene-editing tool with numerous applications in personalized medicine. The CRISPR-Cas mechanism is examined in this review, along with adaptation, crRNA biogenesis, and interference. It also identifies important therapeutic applications in monogenic diseases, cancer immunotherapy, and infectious diseases, as well as pharmacogenetics. There is also discussion of recent developments like neurogenetic editing, programmable gene circuits, and CRISPR-based diagnostics. Enhancing therapeutic efficacy requires the use of CRISPR delivery methods, such as lipid nanoparticles, viral vectors, and non-viral substitutes. Despite its potential, CRISPR technology has drawbacks, including immunogenicity, off-target effects, and ethical issues with germline editing and equitable access.

Keywords: Genome editing technologies (GED), crRNA biogenesis, Neurogenetic editing, sickle cell anemia.

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