Wang G, Sinn P L, McCray P B
Program in Gene Therapy, Department of Pediatrics, University of Iowa College of Medicine, Iowa City, IA 52242, USA.
Curr Opin Mol Ther. 2000 Oct;2(5):497-506.
Retroviral vectors offer several potential advantages for attaining persistent expression of a therapeutic gene in airway epithelia for diseases such as cystic fibrosis. However, several problems have limited their application. Developments in vector production and the advent of lentiviral vectors have increased the investigation of recombinant retrovirus for gene transfer to airway epithelia. In addition, an improved understanding of some of the barriers limiting gene transfer has led to increased transduction efficiencies. The development of novel vector formulations and the use of new envelope pseudotypes are examples of recent findings that are leading to advances in this field.
逆转录病毒载体在实现治疗性基因在气道上皮细胞中持续表达以治疗诸如囊性纤维化等疾病方面具有若干潜在优势。然而,一些问题限制了它们的应用。载体生产方面的进展以及慢病毒载体的出现增加了对重组逆转录病毒用于基因转移至气道上皮细胞的研究。此外,对一些限制基因转移的障碍有了更好的理解,从而提高了转导效率。新型载体配方的开发以及新的包膜假型的使用是导致该领域取得进展的近期研究成果的例子。