Volodina Olga, Smirnikhina Svetlana
Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, 115522, Russia.
Mol Biotechnol. 2025 Feb;67(2):425-437. doi: 10.1007/s12033-024-01070-4. Epub 2024 Feb 16.
The development of gene therapy based on genome editing has opened up new possibilities for the treatment of human genetic disorders. This field has developed rapidly over the past few decades, some genome editing-based therapies are already in phase 3 clinical trials. However, there are several challenges to be addressed before widespread adoption of gene editing therapy becomes possible. The main obstacles in the development of such therapy are safety and efficiency, so one of the biggest issues is the delivery of genetic constructs to patient cells. Approaches in genetic cargo delivery divide into ex vivo and in vivo, which are suitable for different cases. The ex vivo approach is mainly used to edit blood cells, improve cancer therapy, and treat infectious diseases. To edit cells in organs researches choose in vivo approach. For each approach, there is a fairly large set of methods, but, unfortunately, these methods are not universal in their effectiveness and safety. The focus of this article is to discuss the current status of in vivo and ex vivo delivery methods used in genome editing-based therapy. We will discuss the main methods employed in these approaches and their applications in current gene editing treatments under development.
基于基因组编辑的基因治疗发展为人类遗传疾病的治疗开辟了新的可能性。在过去几十年里，这一领域发展迅速，一些基于基因组编辑的疗法已进入3期临床试验。然而，在基因编辑疗法得以广泛应用之前，仍有几个挑战有待解决。这种疗法发展的主要障碍是安全性和有效性，因此最大的问题之一是将基因构建体递送至患者细胞。基因载体递送方法分为体外和体内两种，适用于不同情况。体外方法主要用于编辑血细胞、改善癌症治疗和治疗传染病。为了编辑器官中的细胞，研究人员选择体内方法。对于每种方法，都有相当多的方法，但不幸的是，这些方法在有效性和安全性方面并不通用。本文的重点是讨论基于基因组编辑的疗法中使用的体内和体外递送方法的现状。我们将讨论这些方法中采用的主要方法及其在当前正在开发的基因编辑治疗中的应用。
