کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
1938621 | 1050744 | 2007 | 6 صفحه PDF | دانلود رایگان |
![عکس صفحه اول مقاله: Site-specific transfer of an intact β-globin gene cluster through a new targeting vector Site-specific transfer of an intact β-globin gene cluster through a new targeting vector](/preview/png/1938621.png)
The ideal gene-therapy vector for treating genetic disorders should deliver intact therapeutic genes and their essential regulatory elements into the specific “safe genomic site” and realize long-term, self-regulatory expression. For β-thalassemia gene therapy, viral vectors have been broadly used, but the accompanying insertional mutation and immunogenicity remain problematic. Hence, we aimed to develop new non-viral vectors that are efficient and safe in treating diseases. As previous studies have demonstrated that physiological expression of β-globin genes requires both a 5′ locus control region and 3′ specific elements, we constructed a new human chromosome-derived targeting vector to transfer the intact β-globin gene cluster into K562 cells. The whole β-globin gene cluster was precisely integrated into the target site and expressed in a self-regulatory pattern. The results proved that the human chromosome-derived vector was specifically targeted to the human genome and this could provide a novel platform for further gene therapy research.
Journal: Biochemical and Biophysical Research Communications - Volume 356, Issue 1, 27 April 2007, Pages 32–37