Human Alphoid<sup>tetO</sup> Artificial Chromosome as a Gene Therapy Vector for the Developing Hemophilia A Model in Mice

Human Alphoid<sup>tetO</sup> Artificial Chromosome as a Gene Therapy Vector for the Developing Hemophilia A Model in Mice

Human artificial chromosomes (HACs), including the de novo synthesized alphoid<sup>tetO</sup>-HAC, are a powerful tool for introducing genes of interest into eukaryotic cells. HACs are mitotically stable, non-integrative episomal units that have a large transgene insertion capacity and a...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: MDPI AG 2020-04-01
Series:Cells
Subjects:
human artificial chromosome (HAC)
hemophilia
coagulation factor VIII
alphoid<sup>tetO</sup>-HAC
induced pluripotent stem cells (iPSCs)
microcell-mediated chromosome transfer (MMCT)
Online Access:https://www.mdpi.com/2073-4409/9/4/879

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https://www.mdpi.com/2073-4409/9/4/879

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