­
­
­
­

Inventi Impact: Gene Medicines

Articles

  • Inventi:pgm/31939/20
    Human Alphoid tetO Artificial Chromosome as a Gene Therapy Vector for the Developing Hemophilia a Model in Mice
    Research 2020 : July - September
    Sergey V Ponomartsev, Sergey A Sinenko, Elena V Skvortsova, Mikhail A Liskovykh, Ivan N Voropaev, Maria M Savina, Andrey A Kuzmin, Elena Yu Kuzmina, Alexandra M Kondrashkina, Vladimir Larionov, Natalay Kouprina, Alexey N Tomilin

    Humanartificial chromosomes (HACs), including the de novo synthesized alphoidtetO-HAC,\nare a powerful tool for introducing genes of interest into eukaryotic cells. HACs are mitotically\nstable, non-integrative episomal units that have a large transgene insertion capacity and allow\nefficient and stable transgene expression. Previously, we have shown that the alphoidtetO-HAC\nvector does not interfere with the pluripotent state and provides stable transgene expression in\nhuman induced pluripotent cells (iPSCs) and mouse embryonic stem cells (ESCs). In this study,\nwe have elaborated on a mouse model of ex vivo iPSC- and HAC-based treatment of hemophilia A\nmonogenic disease. iPSCs were developed from....................

    How to Cite this Article
    Attribution/ CC Compliant Citation: Ponomartsev, Sergey V., et al. \"Human AlphoidtetO Artificial Chromosome as a Gene Therapy Vector for the Developing Hemophilia A Model in Mice.\" Cells 9.4 (2020): 879.\nhttps://doi.org/10.3390/cells9040879\nhttp://creativecommons.org/licenses/by/4.0/\nSome formatting elements, header, footer, logos, dates and pagination were modified while adapting this article.
    Download Full Text