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帕金森氏病(Parkinson’s disease,PD)的概念是1817年英国医生James Parkinson提出的.它是发生于中老年期的一种常见的缓慢进展的神经系统退行性疾病,由于黑质多巴胺(DA)能神经无变性造成纹状体系统DA含量下降导致震颤,肌肉僵直、运动迟缓与体位不稳等临床症状.60年代开始的左旋多巴(L-DOPA)替代疗法一直是治疗PD的主流,虽然在一定时间内(3～5a)可明显改善症状,但此法不能减慢疾病的进程,而且随着长期用药和疾病的发展,效果进行性下降,且副作用日趋严重,不能根本解决问题.80年代初开始将自体肾上腺髓质或胚胎黑质细胞移植于脑内,收到一定效果,但细胞来源困难,价格昂贵以及伦理问题限制其在临床的应用.80年代末,随着基因治疗概念的不断深入,基因治疗技术日趋成熟,人们开始了对PD基因治疗的尝试.1989年,Wolff将酪氨酸羟化酶(TH)cDNA转染成纤维细胞,植入实验性PD大鼠脑内,不仅表达出有活性的TH,而且脑内多巴胺的含量明显升高,症状也得到改善,为PD的基因治疗首开先河.
The concept of Parkinson’s disease (PD) was proposed by James Parkinson, a British doctor in 1817. It is a common, slow-progressing neurodegenerative disease that occurs in middle-aged and old age. Since dopamine (DA) Degeneration of the nervous system causes a decrease in DA content in the striatum leading to clinical symptoms such as tremor, muscular stiffness, bradykinesia, and postural instability. L-DOPA replacement therapy started in the 1960s has been the mainstream of PD treatment In a certain period of time (3 ~ 5a) can significantly improve the symptoms, but this method can not slow down the process of disease, and with the long-term medication and disease development, the effect of progressive decline, and the side effects become increasingly serious, can not fundamentally solve the problem.80 Beginning of the year the autologous adrenal medulla or embryonic mesenchymal cells transplanted in the brain, received some results, but the cell source is difficult, expensive and ethical issues limit its clinical application in the late 1980s, with the concept of gene therapy Incessantly, gene therapy technology has matured and people began to try PD gene therapy.In 1989, Wolff transfered tyrosine hydroxylase (TH) cDNA into fibroblasts and implanted experimental PD Rat brain, not only the expression of TH activity out there, but the content of dopamine significantly increased, the symptoms improved gene therapy of PD first of its kind.