Department of Orthopaedics, Shanghai Key Laboratory for Prevention and Treatment of Bone and Joint Diseases, Shanghai Institute of Traumatology and Orthopaedics, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, PR China.
Pharmaceutical Sciences Laboratory, Faculty of Science and Engineering, Åbo Akademi University, Turku, Finland.
Expert Opin Drug Deliv. 2022 Aug;19(8):913-926. doi: 10.1080/17425247.2022.2100342. Epub 2022 Jul 17.
Gene therapy is becoming increasingly common in clinical practice, giving hope for the correction of a wide range of human diseases and defects. The CRISPR/Cas9 system, consisting of the Cas9 nuclease and single-guide RNA (sgRNA), has revolutionized the field of gene editing. However, efficiently delivering the CRISPR-Cas9 to the target organ or cell remains a significant challenge. In recent years, with rapid advances in nanoscience, materials science, and medicine, researchers have developed various technologies that can deliver CRISPR-Cas9 in different forms for and gene editing. Here, we review the development of the CRISPR-Cas9 and describe the delivery forms and the vectors that have emerged in CRISPR-Cas9 delivery, summarizing the key barriers and the promising strategies that vectors currently face in delivering the CRISPR-Cas9.
With the rapid development of CRISPR-Cas9, delivery methods are becoming increasingly important in the delivery of CRISPR-Cas9.
CRISPR-Cas9 is becoming increasingly common in clinical trials. However, the complex nuclease and protease environment is a tremendous challenge for clinical applications. Therefore, the development of delivery methods is highly likely to take the application of CRISPR-Cas9 technology to another level.
基因治疗在临床实践中越来越普遍，为纠正广泛的人类疾病和缺陷带来了希望。由 Cas9 核酸酶和单指导 RNA（sgRNA）组成的 CRISPR/Cas9 系统彻底改变了基因编辑领域。然而，将 CRISPR-Cas9 有效地递送到靶器官或细胞仍然是一个重大挑战。近年来，随着纳米科学、材料科学和医学的快速发展，研究人员开发了各种技术，可将 CRISPR-Cas9 以不同形式递送至不同的目标器官或细胞，用于基因编辑。在此，我们回顾了 CRISPR-Cas9 的发展，并描述了在 CRISPR-Cas9 递送中出现的递送形式和载体，总结了载体在递送 CRISPR-Cas9 时面临的关键障碍和有前途的策略。
随着 CRISPR-Cas9 的快速发展，递送方法在 CRISPR-Cas9 的递送中变得越来越重要。
CRISPR-Cas9 在临床试验中越来越普遍。然而，复杂的核酸酶和蛋白酶环境对临床应用来说是一个巨大的挑战。因此，递送方法的发展很可能将 CRISPR-Cas9 技术的应用提升到另一个水平。
