作为亨廷顿氏病潜在基因治疗靶点的神经营养素。

IF 3.8 4区 医学 Q2 GENETICS & HEREDITY
Sagor Kumar Roy, Ashima Barman, Kumary Labone Sarkar, Seidu A Richard, Bijal Arvinkumar Lacmane
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引用次数: 0

摘要

“亨廷顿氏病”(HD)是一种常染色体显性遗传性神经退行性疾病,其特征是传出纹状体神经元、皮质神经元和基底神经节的缺陷。HD的发病机制尚不清楚,目前还没有治愈这种疾病的治疗方法。本文综述了各种神经营养因子在HD中的潜在有益作用。使用PubMed、Web of Science、Embase和谷歌学者数据库搜索所有关于神经营养因子对HD疗效的研究。几种基因治疗策略已被用于治疗HD,包括多种神经保护因子的基因治疗。此外,多种多样的基因治疗方法,如神经营养因子,在HD的预防和神经保护方面都显示出有希望的结果,这可能是由于它们有可能防止神经元细胞死亡或减少神经退行性变,从而促进创新轴突、树突和突触的生长,从而改善HD。神经营养因子可能适合作为HD的神经保护药物。因此,未来需要进行大量的基因治疗研究,为HD提供更好的治疗选择。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Neurotrophins as Potential Gene Therapy Targets for Huntington's Disease.

"Huntington's disease" (HD) is an autosomal dominant hereditary neurodegenerative disease characterized by defects in efferent striatal neurons, cortical neurons, and the basal ganglia. The pathogenesis of HD is still unclear, and there is currently no curative therapy for this disorder. This review emphasizes the potential beneficial effects of various neurotrophic factors in HD. PubMed, Web of Science, Embase, and google scholar databases were used to search for all studies on the efficacy of neurotrophic factors in HD. Several gene therapy strategies have been employed to treat HD, including gene therapy with a variety of neuroprotective factors. Moreover, a wide variability of gene therapy approaches such as a neurotrophin, has shown promising results for both prevention and neuroprotection in HD, which may be due to their potential to prevent neuronal cell death or decrease neurodegeneration, thereby promoting the growth of innovative axons, dendrites, and synapses leading to improvement of HD. Neurotrophic factors may be suitable as neuroprotective therapy agents in HD. Therefore, substantial research on gene therapy should be conducted to provide better treatment options for HD in the future.

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来源期刊
Current gene therapy
Current gene therapy 医学-遗传学
CiteScore
6.70
自引率
2.80%
发文量
46
期刊介绍: Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases. Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.
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