Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, United States.
Department of Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD 21205, United States.
Curr Gene Ther. 2017;17(3):187-193. doi: 10.2174/1566523217666171013124150.
The recent approval of Spinraza (nusinersen), an antisense oligonucleotide, by U.S. Food and Drug Administration to treat patients with spinal muscular atrophy, has reignited interests of researchers in designing and testing new gene therapy approaches to treat neurological disorders, in particular, to curb neurodegenerative diseases of the central nervous system which represent an ever-increasing public health burden to today's society.
This review highlights several key factors to be taken into consideration to design successful preclinical and clinical gene therapy experiments with respect to the vehicle of delivery and the route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotide therapy and recent clinical trial developments.
美国食品和药物管理局最近批准了反义寡核苷酸药物Spinraza（nusinersen），用于治疗脊髓性肌萎缩症患者，这重新激发了研究人员设计和测试新的基因治疗方法来治疗神经疾病的兴趣，特别是为了抑制中枢神经系统的神经退行性疾病，这些疾病是当今社会日益增长的公共卫生负担。
本综述强调了在设计针对中枢神经系统特定靶点的临床前和临床基因治疗实验时需要考虑的几个关键因素，包括载体的输送和给药途径，另外还重点介绍了反义寡核苷酸治疗和最近的临床试验进展。
