摘要
背景:胶质母细胞瘤是成人中最常见和最具侵袭性的原发性恶性脑肿瘤。尽管其治疗取得了进展,但其预后仍然很差。基因治疗已被提议作为一种补充治疗,因为它可以克服全身治疗的血脑屏障问题,允许局部靶向肿瘤细胞及其肿瘤微环境,而不影响正常的脑实质。与病毒载体相比,非病毒载体因其潜在的生物安全风险降低、成本更低、可用性更高和易于储存而成为一种有吸引力的工具。 目的:在本文中,我们旨在概述用于治疗恶性神经胶质瘤转基因递送的非病毒递送系统的当前临床前和临床发展。 结论:非病毒载体是基因传递的有效工具,因为它们表现出降低的非特异性细胞毒性,并且可以经过多次修饰以获得高肿瘤趋向性和穿过血脑屏障进入肿瘤块的能力。然而,需要在临床前模型和临床试验中进行进一步评估,以便将其转化为神经肿瘤临床。
关键词: 胶质母细胞瘤、非病毒基因治疗、阳离子脂质、树枝状聚合物、树枝状移植物、聚合物胶束、聚(β-氨基酯)、睡美人转座子。
Current Medicinal Chemistry
Title:Current Non-viral Gene Therapy Strategies for the Treatment of Glioblastoma
Volume: 28 Issue: 37
关键词: 胶质母细胞瘤、非病毒基因治疗、阳离子脂质、树枝状聚合物、树枝状移植物、聚合物胶束、聚(β-氨基酯)、睡美人转座子。
摘要:
Background: Glioblastoma constitutes the most frequent and aggressive primary malignant brain tumor in adults. Despite the advances in its treatment, its prognosis remains very poor. Gene therapy has been proposed as a complementary treatment since it may overcome the problem of the blood-brain barrier for systemic therapies, allowing to target tumor cells and their tumor microenvironment locally, without affecting the normal brain parenchyma. In comparison with viral vectors, non-viral vectors became an attractive tool due to their reduced potential of biosafety risks, lower cost, higher availability, and easy storage.
Objective: In this article, we aimed to outline the current preclinical and clinical developments of non-viral delivery systems for therapeutic transgene delivery in malignant gliomas.
Conclusion: Non-viral vectors are efficient tools for gene delivery since they exhibit reduced non-specific cytotoxicity and can go through several modifications in order to achieve high tumor tropism and the ability to cross the blood-brain barrier to access the tumor mass. However, further evaluations in preclinical models and clinical trials are required in order to translate it into the neuro-oncology clinic.
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Cite this article as:
Current Non-viral Gene Therapy Strategies for the Treatment of Glioblastoma, Current Medicinal Chemistry 2021; 28 (37) . https://dx.doi.org/10.2174/0929867328666210525141243
DOI https://dx.doi.org/10.2174/0929867328666210525141243 |
Print ISSN 0929-8673 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-533X |
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