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Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

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초록

Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited andacquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from geneticdisorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explainthe mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilitiesof CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safetyissues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.

Abstract

Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited andacquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from geneticdisorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explainthe mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilitiesof CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safetyissues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.

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