人类疾病转基因小鼠模型在基因治疗研究中的作用

Transgenic Mice of Human Diseases for Gene Therapy

基因治疗一度红红火火，激起公众极大的期望。但是初期试验远不如预期的好。科研人员理智地认识到目前阶段应把研究重点放在更基础的基因结构与功能、基因表达调控及基因载体构建研究等方面。建立人类疾病相关转基因小鼠模型是开展上述研究的一个重要基础。

In the past decade,human gene therapy aspired great expectations from public.However,preliminary studies failed to arrive in the expected goals.Instead,much suspectation aroused.Now scientists realized that they should pay more attention to basic researches,such as gene structures and functions,gene expression and construction of gene vectors.All these studies could be carried out on genetically manipulating animal models of human diseases.This review describes progresses in the generation of transgenic mice as well as a number of potential problems in the construction and utilization of transgenic mice for the study of human diseases.