Therapeutic Applications of Genome Editing Tools for Genetic Diseases
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Since the discovery of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) genome editing technologies, significant efforts have been made to improve their efficiency and accuracy. Consequently, advanced tools such as base editors (BEs) and prime editors (PEs) have emerged over the past decade. Today, CRISPR, BE, and PE systems are actively employed in treating genetic diseases. Notably, CASGEVY by CRISPR therapeutics—the world’s first gene editing treatment using CRISPR technology—received approval from the Food and Drug Administration of both the United Kingdom and the United States last year. This review highlights recent advancements in various CRISPR technologies for therapeutic applications in genetic diseases.
Abstract
Since the discovery of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) genome editing technologies, significant efforts have been made to improve their efficiency and accuracy. Consequently, advanced tools such as base editors (BEs) and prime editors (PEs) have emerged over the past decade. Today, CRISPR, BE, and PE systems are actively employed in treating genetic diseases. Notably, CASGEVY by CRISPR therapeutics—the world’s first gene editing treatment using CRISPR technology—received approval from the Food and Drug Administration of both the United Kingdom and the United States last year. This review highlights recent advancements in various CRISPR technologies for therapeutic applications in genetic diseases.