Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY 10032, USA.
Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory, Columbia University, New York, NY 10032, USA.
Viruses. 2019 Jan 4;11(1):28. doi: 10.3390/v11010028.
The frontiers of precision medicine have been revolutionized by the development of Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR)/Cas9 as an editing tool. CRISPR/Cas9 has been used to develop animal models, understand disease mechanisms, and validate treatment targets. In addition, it is regarded as an effective tool for genome surgery when combined with viral delivery vectors. In this article, we will explore the various viral mechanisms for delivering CRISPR/Cas9 into tissues and cells, as well as the benefits and drawbacks of each method. We will also review the history and recent development of CRISPR and viral vectors and discuss their applications as a powerful tool in furthering our exploration of disease mechanisms and therapies.
CRISPR/Cas9 的发展彻底改变了精准医学的前沿,它是一种编辑工具。CRISPR/Cas9 已被用于开发动物模型、了解疾病机制和验证治疗靶点。此外,当与病毒递送载体结合使用时,它被认为是一种有效的基因组手术工具。在本文中,我们将探讨将 CRISPR/Cas9 递送到组织和细胞中的各种病毒机制,以及每种方法的优缺点。我们还将回顾 CRISPR 和病毒载体的历史和最新发展,并讨论它们作为一种强大工具在进一步探索疾病机制和治疗方法中的应用。