AAV衣壳蛋白基因工程的修饰研究进展

Modify AAV Capsid Protein Via Genetic Engineering

腺相关病毒(AAV)作为载体进行基因治疗已经越来越受人们的青睐,其安全性在帕金森病、囊性纤维病和视网膜疾病等单基因突变疾病临床治疗中得到证明。利用AAV载体进行临床治疗的应用在逐渐增多,提高AAV靶向性和转染效率是人们期盼解决的一道难题。而目前对AAV衣壳蛋白基因工程的修饰,可以明显提高其转导效率和靶向性,一定程度上扫除了其广泛应用AAV的障碍。阐述重组AAV(rAAV)衣壳蛋白在基因工程修饰方面的研究进展及其对基因治疗应用前景的综述。

Adeno-associated virus becomes more and more prevalent as gene therapy vector.The safety was proved in clinical care of Parkinson＇s disease,cystic fibrosis,diseases of retina and so on single-gene diseases.Utilizing AAV vector to clinical care being increasingly.It is a challenge that increases AAV＇s target and transduction.And nowadays modified the AAV capsid protein by genetic engineering can significantly improve the transduction efficiency and targeting.In a certain way to solve the widespread of AAV vector.This review presents the progress of modifying the AAV capsid protein by genetic engineering and this technique is put into use for gene therapy.