Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseases.
Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseases.
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DOI：
10.1002/bies.201400094
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发表时间：
2014-10
期刊：
影响因子：
4
通讯作者：
Isalan, Mark
中科院分区：
文献类型：
--
作者：
Agustin-Pavon, Carmen;Isalan, Mark
合成生物学是一门新兴的工程学科，试图设计和重新连接生物组件，以便以稳健和可预测的方式实现新功能。合成生物学提供的新工具和策略有可能改善神经退行性疾病的治疗方法。特别是，合成生物学将有助于设计小分子、蛋白质、基因网络和载体，以靶向疾病相关基因。最终，新的智能输送系统将提供有针对性和持续的治疗益处。结合“保护和修复”策略将产生新的治疗方法：使用药物治疗，促进神经营养因子合成和基因靶向。除了RNAi和人工转录因子之外，位点特异性基因组修饰可能会发挥越来越大的作用，特别是在新的基因编辑工具如CRISPR/Cas9系统中。总的来说，这些进展将有助于为人类患者的许多脑部疾病开发安全和长期的治疗方法。
Synthetic biology is an emerging engineering discipline that attempts to design and rewire biological components, so as to achieve new functions in a robust and predictable manner. The new tools and strategies provided by synthetic biology have the potential to improve therapeutics for neurodegenerative diseases. In particular, synthetic biology will help design small molecules, proteins, gene networks, and vectors to target disease-related genes. Ultimately, new intelligent delivery systems will provide targeted and sustained therapeutic benefits. New treatments will arise from combining ‘protect and repair’ strategies: the use of drug treatments, the promotion of neurotrophic factor synthesis, and gene targeting. Going beyond RNAi and artificial transcription factors, site-specific genome modification is likely to play an increasing role, especially with newly available gene editing tools such as CRISPR/Cas9 systems. Taken together, these advances will help develop safe and long-term therapies for many brain diseases in human patients.
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DOI：
10.1136/jnnp-2012-304636
发表时间：
2013-10
期刊：
Journal of neurology, neurosurgery, and psychiatry
影响因子：
--
作者：
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