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用于针对病毒感染所致脑损伤的基因治疗的纳米系统。

Nanosystems for gene therapy targeting brain damage caused by viral infections.

作者信息

da Silva Talita Nascimento, de Lima Emanuelle V, Barradas Thaís Nogueira, Testa Carla G, Picciani Paulo H S, Figueiredo Claudia P, do Carmo Flavia A, Clarke Julia R

机构信息

Faculdade de Farmácia, Universidade Federal do Rio de Janeiro, Rio de Janeiro, RJ, 21941-902, Brazil.

Instituto de Ciências Biomédicas, Universidade Federal do Rio de Janeiro, Rio de Janeiro, RJ, 21941-902, Brazil.

出版信息

Mater Today Bio. 2022 Dec 17;18:100525. doi: 10.1016/j.mtbio.2022.100525. eCollection 2023 Feb.

Abstract

Several human pathogens can cause long-lasting neurological damage. Despite the increasing clinical knowledge about these conditions, most still lack efficient therapeutic interventions. Gene therapy (GT) approaches comprise strategies to modify or adjust the expression or function of a gene, thus providing therapy for human diseases. Since recombinant nucleic acids used in GT have physicochemical limitations and can fail to reach the desired tissue, viral and non-viral vectors are applied to mediate gene delivery. Although viral vectors are associated to high levels of transfection, non-viral vectors are safer and have been further explored. Different types of nanosystems consisting of lipids, polymeric and inorganic materials are applied as non-viral vectors. In this review, we discuss potential targets for GT intervention in order to prevent neurological damage associated to infectious diseases as well as the role of nanosized non-viral vectors as agents to help the selective delivery of these gene-modifying molecules. Application of non-viral vectors for delivery of GT effectors comprise a promising alternative to treat brain inflammation induced by viral infections.

摘要

几种人类病原体可导致持久的神经损伤。尽管临床上对这些病症的了解日益增加,但大多数病症仍缺乏有效的治疗干预措施。基因治疗(GT)方法包括改变或调整基因表达或功能的策略,从而为人类疾病提供治疗。由于GT中使用的重组核酸存在物理化学限制且可能无法到达所需组织,因此应用病毒和非病毒载体来介导基因递送。尽管病毒载体与高水平的转染相关,但非病毒载体更安全且已得到进一步探索。由脂质、聚合物和无机材料组成的不同类型的纳米系统被用作非病毒载体。在本综述中,我们讨论了GT干预的潜在靶点,以预防与传染病相关的神经损伤,以及纳米级非病毒载体作为帮助选择性递送这些基因修饰分子的药物的作用。应用非病毒载体递送GT效应物是治疗病毒感染引起的脑部炎症的一种有前景的替代方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74ad/9816812/cb9c6b15d6da/ga1.jpg

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