Genetically改良遗传性疾病动物模型用于基因导向治疗试验

Q3 Pharmacology, Toxicology and Pharmaceutics
A. Polikarpova, T. Egorova, M. Bardina
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引用次数: 1

摘要

致病基因已被鉴定为许多严重的肌肉和神经遗传疾病。基因治疗领域的进展为治疗这些危及生命的疾病的药物开发提供了有前景的解决方案。根据突变如何影响基因产物的功能,不同的基因治疗方法可能是有益的。基因替代疗法适用于由导致功能蛋白缺乏的突变引起的疾病。对于由突变基因的毒性产物引起的疾病,建议采用基因抑制策略。剪接调节剂、基因组编辑和碱基编辑技术可以应用于具有不同类型潜在突变的疾病。在人类疾病的动物模型中测试潜在药物是开发中不可或缺的一步。考虑到特定的基因治疗方法,可以使用从转基因到精确基因组编辑的各种技术生成合适的动物模型。在这篇综述中,我们讨论了用于生成最常见的肌肉和神经遗传疾病的小型和大型动物模型的技术。我们特别关注用于测试基于腺相关载体和反义核苷酸的基因治疗的动物模型。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Genetically modified animal models of hereditary diseases for testing of gene-directed therapy
Disease-causing genes have been identified for many severe muscular and neurological genetic disorders. Advances in the gene therapy field offer promising solutions for drug development to treat these life-threatening conditions. Depending on how the mutation affects the function of the gene product, different gene therapy approaches may be beneficial. Gene replacement therapy is appropriate for diseases caused by mutations that result in the deficiency of the functional protein. Gene suppression strategy is suggested for disorders caused by the toxic product of the mutant gene. Splicing modulators, genome editing, and base editing techniques can be applied to disorders with different types of underlying mutations. Testing potential drugs in animal models of human diseases is an indispensable step of development. Given the specific gene therapy approach, appropriate animal models can be generated using a variety of technologies ranging from transgenesis to precise genome editing. In this review, we discuss technologies used to generate small and large animal models of the most common muscular and neurological genetic disorders. We specifically focus on animal models that were used to test gene therapies based on adeno-associated vectors and antisense nucleotides.
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来源期刊
Research Results in Pharmacology
Research Results in Pharmacology Medicine-Pharmacology (medical)
CiteScore
1.50
自引率
0.00%
发文量
32
审稿时长
12 weeks
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