Wallenberg Neuroscience Center, Department of Experimental Medical Science, Lund University, Lund, Sweden.
Sci Transl Med. 2009 Oct 14;1(2):2ps2. doi: 10.1126/scitranslmed.3000350.
The introduction of L-dopa (L-3,4-dihydroxyphenylalanine) therapy 40 years ago was a revolution in the treatment of patients with Parkinson s disease (PD). With time, however, the shortcomings of oral L-dopa medication became apparent, in particular the appearance of troublesome side effects, expressed as involuntary movements (dyskinesias) that developed over time in many patients. A gene therapy approach, aimed at restoring dopamine synthesis in the affected brain by viral vector delivery of genes that encode the dopamine-synthesizing enzymes, may offer a solution to this problem. Now, a team of French and UK researchers reports promising results in a nonhuman primate model of PD, paving the way for clinical trials of this enzyme-replacement approach.
40 年前左旋多巴（L-3,4-二羟基苯丙氨酸）疗法的引入是帕金森病（PD）治疗领域的一场革命。然而，随着时间的推移，口服左旋多巴药物的缺点变得明显，特别是出现了麻烦的副作用，表现为许多患者随时间推移出现的不自主运动（运动障碍）。一种基因治疗方法，通过病毒载体传递编码多巴胺合成酶的基因，旨在恢复受影响大脑中的多巴胺合成，可能为解决这个问题提供了一种解决方案。现在，一个法英研究小组在 PD 的非人类灵长类动物模型中报告了有希望的结果，为这种酶替代方法的临床试验铺平了道路。
