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用于肺定向基因治疗的腺相关病毒和慢病毒假型。

Adeno-associated virus and lentivirus pseudotypes for lung-directed gene therapy.

作者信息

Wilson James M

机构信息

204 Wistar Institute, 3601 Spruce Street, Philadelphia, PA 19104-4268, USA.

出版信息

Proc Am Thorac Soc. 2004;1(4):309-14. doi: 10.1513/pats.200409-041MS.

Abstract

The enthusiasm for cystic fibrosis gene therapy that attended the initial cloning of the gene and in vitro correction of the genetic defect eventually diminished as we learned more about the limitations of vector technologies that were available in the 1980s and 1990s. Substantial progress has been made, however, over the last 5 years in developing second- and third-generation vector constructs that should be more useful in achieving gene transfer to the lung for the treatment of pulmonary diseases such as cystic fibrosis.

摘要

随着我们对20世纪80年代和90年代可用载体技术的局限性有了更多了解,最初克隆该基因并在体外纠正基因缺陷时对囊性纤维化基因治疗的热情最终有所消退。然而,在过去5年中,第二代和第三代载体构建体的开发取得了重大进展,这些构建体在实现向肺部进行基因转移以治疗诸如囊性纤维化等肺部疾病方面应该会更有用。

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