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Advances in CRISPR Delivery Methods: Perspectives and Challenges
The CRISPR Journal ( IF 3.7 ) Pub Date : 2022-10-13 , DOI: 10.1089/crispr.2022.0051
Selami Demirci 1 , Khaled Essawi 1, 2 , Paula Germino-Watnick 1 , Xiong Liu 1 , Waleed Hakami 2 , John F Tisdale 1
Affiliation  

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.

中文翻译:

CRISPR 递送方法的进展:前景和挑战

随着新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校正即将出现。理想情况下,基因组编辑方法将通过永久性疾病纠正而不是简单地对症治疗患者来提供长期解决方案。尽管存在各种编辑机制选项,但由于其高编辑效率、简单性和经济性,成簇规律间隔的短回文重复序列 (CRISPR)-Cas(CRISPR 相关蛋白)编辑技术已成为最受欢迎的编辑技术。然而,尽管 CRISPR 技术正在逐渐完善,但如果无法将其有效、安全地递送至所需的细胞或组织,优化将是徒劳的。所以,重要的是,科学家们同时也要专注于发明和改进编辑机器的交付方式。在这篇综述中,我们将讨论病毒和非病毒递送系统的关键细节,包括有效载荷、免疫原性、递送功效、临床应用和未来方向。
更新日期:2022-10-13
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