亨廷顿疾病是一种神经退化疾病，这类疾病的部分特征是纹状体多棘投射神经元减少（MSNs）。Benraiss研究团队在这一疾病的治疗领域获得了突破。

他们通过脑室内注射带有BDNF和noggin的腺病毒，发现引发了新纹状体中新神经元的产生。单次脑室内注射AAV4-BDNF和AAV4-noggin腺病毒能引发长期募集新的MSNs，而这种反应不管在野生型小鼠还是亨廷顿动物模型R6/2中都是一致的。直接BDNF和noggin蛋白处理与脑室内注射AAV4-BDNF和AAV4-noggin腺病毒的结果是一致的，也能活化招募室管膜下前体细胞从而形成新的MSNs，这些细胞进一步成熟整合进入神经环路中。更重要的是，AAV4-BDNF/noggin处理的R6/2 小鼠表现出运动功能衰退得到缓解，存活进一步增加。

在松鼠猴给予脑室内注射BDNF/noggin表现出相似的纹状体神经元的增加，这项研究可能对亨廷顿疾病指出了新的治疗策略。

原文摘要：

Sustained Mobilization of Endogenous Neural Progenitors Delays Disease Progression in a Transgenic Model of Huntington’s Disease

Huntington’s disease (HD) is a neurodegenerative disease characterized in part by the loss of striatopallidal medium spiny projection neurons (MSNs). Expression of BDNF and noggin via intracerebroventricular (ICV) delivery in an adenoviral vector triggers the addition of new neurons to the neostriatum. In this study, we found that a single ICV injection of the adeno-associated viruses AAV4-BDNF and AAV4-noggin triggered the sustained recruitment of new MSNs in both wild-type and R6/2 mice, a model of HD. Mice treated with AAV4-BDNF/noggin or with BDNF and noggin proteins actively recruited subependymal progenitor cells to form new MSNs that matured and achieved circuit integration. Importantly, the AAV4-BDNF/noggin-treated R6/2 mice showed delayed deterioration of motor function and substantially increased survival. In addition, squirrel monkeys given ICV injections of adenoviral BDNF/noggin showed similar addition of striatal neurons. Induced neuronal addition may therefore represent a promising avenue for disease amelioration in HD.