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肽穿梭载体介导的脑基因治疗。

Peptide Shuttle-Mediated Delivery for Brain Gene Therapies.

机构信息

Institute for Research in Biomedicine (IRB Barcelona), The Barcelona Institute of Science and Technology (BIST), Barcelona 08028, Spain.

出版信息

Curr Top Med Chem. 2020;20(32):2945-2958. doi: 10.2174/1568026620666201023122903.

Abstract

The manipulation of an individual's genetic information to treat a disease has revolutionized the biomedicine field. Despite the promise of gene therapy, this treatment can have long-term sideeffects. Efforts in the field and recent discoveries have already led to several improvements, including efficient gene delivery and transfer, as well as inpatient safety. Several studies to treat a wide range of pathologies-such as cancer or monogenic diseases- are currently being conducted. Here we provide a broad overview of methodologies available for gene therapy, placing a strong emphasis on treatments for central nervous system diseases. Finally, we give a perspective on current delivery strategies to treat such diseases, with a special focus on systems that use peptides as delivery vectors.

摘要

个体遗传信息的操纵被用来治疗疾病,这一技术已经彻底改变了生物医学领域。尽管基因治疗前景广阔,但这种治疗方法可能会产生长期的副作用。该领域的努力和最近的发现已经带来了一些改进,包括高效的基因传递和转移,以及住院安全性。目前正在进行多项研究来治疗各种病理疾病,如癌症或单基因疾病。在这里,我们提供了基因治疗可用方法的广泛概述,特别强调了中枢神经系统疾病的治疗方法。最后,我们对当前用于治疗此类疾病的传递策略提出了看法,特别关注使用肽作为传递载体的系统。

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