神经退行性疾病基因治疗方法的挑战和进展

Title:Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders

Volume: 17 Issue: 3

关键词: 基因治疗，神经退行性疾病，反义寡核苷酸，临床试验，病毒载体，中枢神经系统。

摘要: Introduction: 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.

Conclusion: 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.

Cite this article as:

Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders, Current Gene Therapy 2017; 17 (3) . https://dx.doi.org/10.2174/1566523217666171013124150