用于基因治疗的神经疾病大型动物模型。
Large animal models of neurological disorders for gene therapy.
作者信息
Gagliardi Christine, Bunnell Bruce A
机构信息
Tulane National Primate Research Center, Covington, LA 70433-8915, USA.
出版信息
ILAR J. 2009;50(2):128-43. doi: 10.1093/ilar.50.2.128.
he development of therapeutic interventions for genetic disorders and diseases that affect the central nervous system (CNS) has proven challenging. There has been significant progress in the development of gene therapy strategies in murine models of human disease, but gene therapy outcomes in these models do not always translate to the human setting. Therefore, large animal models are crucial to the development of diagnostics, treatments, and eventual cures for debilitating neurological disorders. This review focuses on the description of large animal models of neurological diseases such as lysosomal storage diseases, Parkinsons disease, Huntingtons disease, and neuroAIDS. The review also describes the contributions of these models to progress in gene therapy research.
事实证明，开发针对影响中枢神经系统（CNS）的遗传疾病和病症的治疗干预措施具有挑战性。在人类疾病的小鼠模型中，基因治疗策略的开发取得了重大进展，但这些模型中的基因治疗结果并不总是能转化到人类环境中。因此，大型动物模型对于衰弱性神经疾病的诊断、治疗及最终治愈的发展至关重要。本综述着重描述神经疾病的大型动物模型，如溶酶体贮积病、帕金森病、亨廷顿病和神经艾滋病。该综述还描述了这些模型对基因治疗研究进展的贡献。
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