转基因治疗根性撕脱以及脊髓损伤的研究进展（英文）

Gene therapy for root avulsion and spinal cord injuries

根性撕脱伤和脊髓损伤是临床上非常重要的疾病,临床治疗方法很多,但难以成功治愈根性撕脱和脊髓损伤引起的瘫痪或慢性疼痛.基因治疗有可能为有效地治疗根性撕脱伤和脊髓损伤,并减轻对病人、家庭、医疗机构和世界经济的影响.癌症的治疗中基因治疗已解决了临床许多挑战性的问题,靶点基因的脊髓损伤研究从实验室走向临床.本综述从臂丛神经撕脱伤和脊髓损伤相关的血脊髓屏障的解剖和病理表现,脊髓损伤和根性撕脱伤基因治疗的种类,以及治疗脊髓损伤活体动物应用到的基因转运载体的种类,重点阐述病毒和非病毒载体转基因治疗在脊髓的转运路径.靶向脊髓的基因治疗已有很长时间,但仍然有问题没有解决,尤其是如何有效地转导病毒载体入在体动物的神经元仍未清楚.在病毒载体的安全性和设计上的问题未解决之前,临床治疗尚需进一步研究.

Root avulsion and spinal cord injuries are very important clinical entities that have vexed scientists and clinicians for a long time.Many treatments have been designed and applied clinically but none of them has successfully cured paralysis or chronic pain that comes with these conditions in the clinical area.In pursuit of a solution to root avulsion and spinal cord injuries,gene therapy has emerged as a powerful tool that could potentially mitigate the impact on patients,families,healthcare resources and world economies.Clinical trials involving gene therapy in other conditions such as cancer have unravelled a lot of promises and challenges that have served as pointers to guide the imminent scaling up of spinal cord targeted gene therapy from the experimental work to everyday clinical utility.This review discusses the anatomy and the brachial plexus root avulsion of and spinal cord injuries-related pathobehaviour of the blood spinal cord barrier,types of gene therapy and the gene cargo delivery vectors currently being exploited in the management of spinal cord injuries.Major highlights of the viral and non viral gene delivery routes are explored while pointing out areas where they have been found wanting warranting much more exploration.It is clear that gene therapy targeted at the spinal cord has come a long way and still has more basic science research needed especially for the potentially transduction-efficient viral vectors.Until the safety and design issues raised about the viral vectors are taken care of,clinical translation will remain a dream at the horizon.