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保护并非总是好事：免疫系统对基因治疗的影响。

Protection is not always a good thing: The immune system's impact on gene therapy.

作者信息

Freitas Martiela Vaz de, Frâncio Lariane, Haleva Laura, Matte Ursula da Silveira

机构信息

Hospital de Clínicas de Porto Alegre, Centro de Pesquisa Experimental, Laboratório Células Tecidos e Genes, Porto Alegre, RS, Brazil.

Universidade Federal do Rio Grande do Sul, Programa de Pós-Graduação em Genética e Biologia Molecular, Porto Alegre, RS, Brazil.

出版信息

Genet Mol Biol. 2022 Jul 15;45(3 Suppl 1):e20220046. doi: 10.1590/1678-4685-GMB-2022-0046. eCollection 2022.

DOI:10.1590/1678-4685-GMB-2022-0046
PMID:35852088
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9295005/
Abstract

There are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the efficiency of gene therapy in different ways. Here we present an overview of approved gene therapy products and the immune response elicited by gene delivery systems. These include responses against the vector or its content after delivery and against the product of the corrected gene. Strategies to overcome the hurdles include hiding the vector or/and the transgene product from the immune system and hiding the immune system from the vector/transgene product. Combining different strategies, such as patient screening and intelligent vector design, gene therapy is set to make a difference in the life of patients with severe genetic diseases.

摘要

目前有许多针对基因疗法开发的临床试验，其中一些已经使基因治疗产品获得了商业批准。在开发更安全、更有效的基因传递和调控策略方面取得了重大进展。一个尚未解决的方面是免疫系统，它会以不同方式影响基因治疗的效率。在此，我们概述已批准的基因治疗产品以及基因传递系统引发的免疫反应。这些反应包括在基因传递后针对载体或其内容物以及针对校正基因产物的反应。克服这些障碍的策略包括使载体或/和转基因产物躲避免疫系统以及使免疫系统躲避载体/转基因产物。通过结合不同策略，如患者筛选和智能载体设计，基因疗法必将改变重症遗传病患者的生活。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/514a8dba8eda/1415-4757-GMB-45-03-s1-e20220046-gf5.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/8d1c6605cb97/1415-4757-GMB-45-03-s1-e20220046-gf1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/0634b3c7c7d0/1415-4757-GMB-45-03-s1-e20220046-gf2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/aa8d020320d4/1415-4757-GMB-45-03-s1-e20220046-gf3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/de3a500b51b1/1415-4757-GMB-45-03-s1-e20220046-gf4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/514a8dba8eda/1415-4757-GMB-45-03-s1-e20220046-gf5.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/8d1c6605cb97/1415-4757-GMB-45-03-s1-e20220046-gf1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/0634b3c7c7d0/1415-4757-GMB-45-03-s1-e20220046-gf2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/aa8d020320d4/1415-4757-GMB-45-03-s1-e20220046-gf3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/de3a500b51b1/1415-4757-GMB-45-03-s1-e20220046-gf4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/94ff/9295005/514a8dba8eda/1415-4757-GMB-45-03-s1-e20220046-gf5.jpg

相似文献

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[3]
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[5]
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[6]
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[7]
Immunogenicity of CRISPR therapeutics-Critical considerations for clinical translation.

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本文引用的文献

[1]
Structural Analysis of Retrovirus Assembly and Maturation.

Viruses. 2021-12-29

[2]
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Blood Cancer J. 2021-4-6

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