Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, Maryland, USA; and College of Applied Medical Sciences, Jazan University, Jazan, Saudi Arabia.
Department of Medical Laboratory Science, College of Applied Medical Sciences, Jazan University, Jazan, Saudi Arabia.
CRISPR J. 2022 Oct;5(5):660-676. doi: 10.1089/crispr.2022.0051.
With the advent of new genome editing technologies and the emphasis placed on their optimization, the genetic and phenotypic correction of a plethora of diseases sit on the horizon. Ideally, genome editing approaches would provide long-term solutions through permanent disease correction instead of simply treating patients symptomatically. Although various editing machinery options exist, the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas (CRISPR-associated protein) editing technique has emerged as the most popular due to its high editing efficiency, simplicity, and affordability. However, while CRISPR technology is gradually being perfected, optimization is futile without accessible, effective, and safe delivery to the desired cell or tissue. Therefore, it is important that scientists simultaneously focus on inventing and improving delivery modalities for editing machinery as well. In this review, we will discuss the critical details of viral and nonviral delivery systems, including payload, immunogenicity, efficacy in delivery, clinical application, and future directions.
随着新的基因组编辑技术的出现以及对其优化的重视,大量疾病的遗传和表型纠正即将成为现实。理想情况下,基因组编辑方法将通过永久性疾病纠正提供长期解决方案,而不仅仅是对症治疗患者。虽然存在各种编辑机制选择,但由于其高效率、简单性和可负担性,簇状规则间隔短回文重复 (CRISPR)-Cas(CRISPR 相关蛋白)编辑技术已成为最受欢迎的技术。然而,尽管 CRISPR 技术在逐渐完善,但如果无法将其有效地递送到所需的细胞或组织中,那么优化也是徒劳的。因此,科学家们同时关注发明和改进编辑机制的传递方式非常重要。在这篇综述中,我们将讨论病毒和非病毒传递系统的关键细节,包括有效载荷、免疫原性、传递效率、临床应用和未来方向。