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迈向神经系统的基因治疗。

Towards gene therapy in the nervous system.

作者信息

Doering L C

机构信息

Health Science Center, McMaster University, Hamilton, Ontario, Canada.

出版信息

Clin Neurosci. 1995;3(5):259-61.

PMID:8914791
Abstract

The integration of gene transfer techniques with neuroscience provides new opportunities to study the cellular and molecular aspects of neuron survival, dysfunction, and degeneration. A variety of viral vectors have been used to probe the effects of new gene expression in neurons, glial cells, and progenitor cells. These vectors provide an avenue to deliver genes with potential therapeutic value as shown by neuroprotective effects in animal models subsequent to the transduction of selected cell types. In addition, basic research in this field provides insight into the signalling mechanisms that dictate cell survival and programmed cell death and helps shape future strategies for gene therapy in the central nervous system. New generations of viral vectors will offer improved gene expression and move the field of gene delivery/therapy closer to clinical reality for the prevalent neurodegenerative conditions that include Alzheimer's and Parkinson's.

摘要

基因转移技术与神经科学的结合为研究神经元存活、功能障碍和退化的细胞及分子层面提供了新机遇。多种病毒载体已被用于探究新基因在神经元、胶质细胞和祖细胞中的表达效果。这些载体为传递具有潜在治疗价值的基因提供了一条途径,如在选定细胞类型转导后动物模型中的神经保护作用所示。此外,该领域的基础研究有助于深入了解决定细胞存活和程序性细胞死亡的信号机制,并有助于塑造未来中枢神经系统基因治疗的策略。新一代病毒载体将提供更好的基因表达,并使基因递送/治疗领域更接近包括阿尔茨海默病和帕金森病在内的常见神经退行性疾病的临床应用。

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