基因治疗的研究进展

Research Advances in Gene Therapy

目的 :基因治疗人类单基因遗传病和癌症。方法 :阅读国外文献 ,总结分析了基因治疗的临床研究。结果 :数百例研究证明基因治疗都有疗效 ,副作用极少 ,存在的问题是在体内基因导入和表达效率不理想。结论 :有待开发高效的基因表达载体系统和受体细胞体系 ;

Objective:Aim to treat human monogenic inherited diseases and cancers.Methods:Reading the recent literatueres abroad,a series of clinical trials of gene therapy for inherited diseases and cancers were analysed and summured.Results:Hundreds trials showed that gene therapy has the potential effects,with a low risk of adverse reaction,but the efficiency of gene transfer and expression in vivo is still disappointingly low.Conclusion:We suggested that highly efficient gene expression systems and receptor cell systems were might be further developed;Therapeutic cloning technique may be a powerful approach which resolve above problems.