摘要
简介:美国食品及药物管理局(FDA)最近批准Spinraza(nusinersen)用于治疗脊髓性肌肉萎缩症患者,这引发了研究人员重新设计和测试治疗神经系统疾病的新基因治疗方法的兴趣,以遏制中枢神经系统的神经退行性疾病,这是今天社会日益增加的公共卫生负担。 结论:本综述强调了几个要考虑的关键因素,以便设计成功的临床前和临床基因治疗实验的运载工具和给予中枢神经系统特异性靶点的途径,另外重点关注反义寡核苷酸治疗和近期临床试验进展。
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。
Current Gene Therapy
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.
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Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders, Current Gene Therapy 2017; 17 (3) . https://dx.doi.org/10.2174/1566523217666171013124150
DOI https://dx.doi.org/10.2174/1566523217666171013124150 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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