摘要
血友病是一种单基因遗传性疾病,主要发病机制为编码凝血因子的基因突变所导致凝血因子减少。临床表现为反复出血,严重时可危及生命,尚不能治愈。目前正在研究的基因治疗则具有治愈血友病的可能。在基因治疗的研究中,动物模型是必不可少的基础支持条件。血友病鼠模型因其自身特点,如便于运输,研究费用少于其它大型动物等,在众多动物模型中脱颖而出,成为血友病动物模型研究的热点。血友病鼠模型的构建经过20多年的历程,逐渐成熟。极大地促进了血友病基因治疗的发展,现就血友病鼠的构建及研究进展予以综述。
Hemophilia is a monogenic hereditary disease whose pathogenesis includes the reduction of coagulation factors caused by mutations in genes encoding coagulation factors.The clinical manifestation of this disease is recurrent bleeding,which can be life-threatening in severe cases and cannot be cured.Currently,gene therapy might have the potential to cure hemophilia.In gene therapy research,animal models are commonly used.The mouse hemophilia model is useful because of its convenience for transportation and lower research costs when compared with other large animal models.It has become a hot spot for research using animal hemophilia models.After more than 20 years of development,technology to generate a mouse hemophilia model has gradually matured.The aim of this paper is to summarize the research progress in generation of a mouse hemophilia model.
作者
李杰
闫振宇
LI Jie;YAN Zhenyu(North China University of Science and Technology,Tangshan 063000,China;The Affiliated Hospital of North China University of Science and Technology,Tangshan 063000)
出处
《中国比较医学杂志》
CAS
北大核心
2019年第6期141-146,共6页
Chinese Journal of Comparative Medicine
基金
“2016年河北省政府临床医学优秀人才培养和基础课题研究项目”(361036)