Institute for Research in Biomedicine (IRB Barcelona), The Barcelona Institute of Science and Technology (BIST), Barcelona 08028, Spain.
Curr Top Med Chem. 2020;20(32):2945-2958. doi: 10.2174/1568026620666201023122903.
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.
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。
