A TRANSGENIC MONKEY: INHERITED NEURODEGENERATIVE DISEASES

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者的研究机构。 非人灵长类动物（NHPs;猴子）在人类医学研究的许多领域做出了重大贡献，因为它们是研究人类疾病的宝贵模型。近年来，随着猴基因组信息和转基因技术的发展，首次建立了亨廷顿病（HD）转基因猴模型。转基因HD猴出现与人类患者相似的细胞变化和神经病理学，这在啮齿动物模型中很少观察到。除了突变亨廷顿蛋白（htt）对猴子的神经毒性外，HD猴子还出现与HD患者相似的不自主运动和协调身体运动的困难。我们已经成功地实现了我们的目标，在开发一个转基因的HD猴，表达突变htt和发展的症状相当于人类患者的原始提案。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Nonhuman primates (NHPs; monkeys) have contributed significantly in many areas of human medicine research as they are invaluable models for studying human diseases. Recent advancements in monkey genomic information and transgenic technology have resulted in the creation of the first transgenic monkey model of Huntington's disease (HD). Transgenic HD monkeys develop cellular changes and neuropathologies similar to that of human patients, which are rarely observed in rodent models. Besides the neurotoxicity of mutant huntingtin (htt) in monkeys, HD monkeys also develop involuntary movement and difficulties in coordinating body movement similar to that of HD patients. We have successfully accomplished our goal of the original proposal in developing a transgenic HD monkey that expresses mutant htt and develops symptoms comparable to human patients.