Laboratory of Genetic Technologies, Martsinovsky Institute of Medical Parasitology, Tropical and Vector-Borne Diseases, First Moscow State Medical University (Sechenov University), 119991 Moscow, Russia.
Division of Biotechnology, Sirius University of Science and Technology, 354340 Sochi, Russia.
Cells. 2024 Nov 19;13(22):1916. doi: 10.3390/cells13221916.
Gene therapies hold significant promise for treating previously incurable diseases. A number of gene therapies have already been approved for clinical use. Currently, gene therapies are mostly limited to the use of adeno-associated viruses and the herpes virus. Viral vectors, particularly those derived from human viruses, play a critical role in this therapeutic approach due to their ability to efficiently deliver genetic material to target cells. Despite their advantages, such as stable gene expression and efficient transduction, viral vectors face numerous limitations that hinder their broad application. These limitations include small cloning capacities, immune and inflammatory responses, and risks of insertional mutagenesis. This review explores the current landscape of viral vectors used in gene therapy, discussing the different types of DNA- and RNA-based viral vectors, their characteristics, limitations, and current medical and potential clinical applications. The review also highlights strategies to overcome existing challenges, including optimizing vector design, improving safety profiles, and enhancing transgene expression both using molecular techniques and nanotechnologies, as well as by approved drug formulations.
基因疗法在治疗以前无法治愈的疾病方面具有重要的前景。已经有许多基因疗法被批准用于临床使用。目前，基因疗法主要限于使用腺相关病毒和单纯疱疹病毒。病毒载体，特别是那些源自人类病毒的载体，在这种治疗方法中起着至关重要的作用，因为它们能够将遗传物质有效地递送到靶细胞。尽管它们具有稳定的基因表达和高效转导等优势，但病毒载体面临着许多限制，阻碍了它们的广泛应用。这些限制包括较小的克隆容量、免疫和炎症反应以及插入突变的风险。这篇综述探讨了基因治疗中使用的病毒载体的现状，讨论了不同类型的基于 DNA 和 RNA 的病毒载体，它们的特点、局限性以及当前的医学和潜在的临床应用。该综述还强调了克服现有挑战的策略，包括优化载体设计、提高安全性、利用分子技术和纳米技术以及经过批准的药物制剂来增强转基因表达。
