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帕金森病的基因疗法

Gene-Based Therapeutics for Parkinson's Disease.

作者信息

Shalaby Karim E, El-Agnaf Omar M A

机构信息

Biological and Biomedical Sciences Division, College of Health & Life Sciences (CHLS), Hamad Bin Khalifa University (HBKU), Qatar Foundation, Doha 34110, Qatar.

Neurological Disorders Research Center, Qatar Biomedical Research Institute (QBRI), Hamad Bin Khalifa University (HBKU), Qatar Foundation, Doha 34110, Qatar.

出版信息

Biomedicines. 2022 Jul 26;10(8):1790. doi: 10.3390/biomedicines10081790.

Abstract

Parkinson's disease (PD) is a complex multifactorial disorder that is not yet fully surmised, and it is only when such a disease is tackled on multiple levels simultaneously that we should expect to see fruitful results. Gene therapy is a modern medical practice that theoretically and, so far, practically, has demonstrated its capability in joining the battle against PD and other complex disorders on most if not all fronts. This review discusses how gene therapy can efficiently replace current forms of therapy such as drugs, personalized medicine or invasive surgery. Furthermore, we discuss the importance of enhancing delivery techniques to increase the level of transduction and control of gene expression or tissue specificity. Importantly, the results of current trials establish the safety, efficacy and applicability of gene therapy for PD. Gene therapy's variety of potential in interfering with PD's pathology by improving basal ganglial circuitry, enhancing dopamine synthesis, delivering neuroprotection or preventing neurodegeneration may one day achieve symptomatic benefit, disease modification and eradication.

摘要

帕金森病(PD)是一种复杂的多因素疾病,目前尚未完全明了,只有当这种疾病在多个层面同时得到解决时,我们才有望看到丰硕的成果。基因治疗是一种现代医学实践,从理论上讲,到目前为止,实际上已经证明了它在参与对抗帕金森病和其他复杂疾病的战斗中,在大多数甚至所有方面都具有作战能力。这篇综述讨论了基因治疗如何能够有效地替代当前的治疗形式,如药物、个性化医疗或侵入性手术。此外,我们还讨论了增强递送技术以提高转导水平以及控制基因表达或组织特异性的重要性。重要的是,当前试验的结果证实了基因治疗对帕金森病的安全性、有效性和适用性。基因治疗通过改善基底神经节回路、增强多巴胺合成、提供神经保护或预防神经退行性变来干扰帕金森病病理的多种潜力,也许有一天能够实现症状改善、疾病修饰和根除。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/50bc/9331241/3922fdbc7849/biomedicines-10-01790-g001.jpg

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