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.

操纵个体的遗传信息以治疗疾病彻底改变了生物医学领域。尽管有基因治疗的希望，但这种治疗可能会产生长期的副作用。在该领域的努力和最近的发现已经带来了一些改进，包括有效的基因传递和转移以及住院安全性。目前正在进行数种研究，以治疗各种病理学，例如癌症或单基因疾病。在这里，我们提供了可用于基因治疗的方法的广泛概述，重点放在中枢神经系统疾病的治疗上。最后，我们对目前用于治疗此类疾病的递送策略给出了看法，特别关注使用肽作为递送载体的系统。