Key result
CRISPR-based genome editing shows therapeutic potential for genetic disorders despite remaining safety and efficacy issues.
Why the study?
Tools to correct pathogenic genetic variants are rapidly evolving, motivating an examination of CRISPR-based genome editing principles, new editor mechanisms, and therapeutic possibilities from recent clinical trials and animal studies.
Answers are grounded in this paper and cite their sources.
This review outlines the basic principles and emerging clinical applications of CRISPR-based genome editing, highlighting its potential to treat genetic disorders despite ongoing safety and efficacy concerns.
Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.
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Lim et al. (2022) conducted a review in Genetic disorders. CRISPR-based genome editing was evaluated. CRISPR-based genome editing technologies show significant therapeutic potential for treating genetic disorders in ongoing clinical trials and animal models, though efficacy and safety issues remain.
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