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Life sciences · Journal article
Nucleosides Nucleotides & Nucleic Acids · September 29, 2026
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CRISPR-Cas9 has entered the mainstream of genetic research and therapeutic development, offering a level of programmable and targeted gene editing that has substantially expanded the possibilities of genetic manipulation. This review focuses on the discovery to development of CRISPR-Cas9 and its recent practice in pharmacological and biotechnology field. We discuss further advancements in CRISPR technology such as high-fidelity CRISPR and new systems Cas12 and Cas13 which increases efficiency of editing and opens more opportunities. CRISPR technology has been further advanced when combined with Artificial Intelligence and Machine Learning; guide RNA is now optimized while the off-target effects are predicted. The review also focuses on the new targets for therapy, recent trials, the legal and the social and ethical relevance of the CRISPR approach. With the development of CRISPR-Cas9 promising and revolutionary preliminary achievements in the fields of drug development and disease management and tissue engineering are anticipated. Although, in its application, there are questions that have been raised concerning side effects, carriers, and ethics, it is important to take advantage of it in the right manner. As seen in this review, CRISPR-Cas9 plays the crucial part of the development of biomedical studies and therapeutic paradigms.