Kazuki, Y;
Uno, N;
Abe, S;
Kajitani, N;
Kazuki, K;
Yakura, Y;
Sawada, C;
... Tomizuka, K; + view all
(2021)
Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling.
Molecular Therapy: Nucleic Acids
, 23
pp. 629-639.
10.1016/j.omtn.2020.12.012.
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Abstract
Genetic engineering of induced pluripotent stem cells (iPSCs) holds great promise for gene and cell therapy as well as drug discovery. However, there are potential concerns regarding the safety and control of gene expression using conventional vectors such as viruses and plasmids. Although human artificial chromosome (HAC) vectors have several advantages as a gene delivery vector, including stable episomal maintenance and the ability to carry large gene inserts, the full potential of HAC transfer into iPSCs still needs to be explored. Here, we provide evidence of a HAC transfer into human iPSCs by microcell-mediated chromosome transfer via measles virus envelope proteins for various applications, including gene and cell therapy, establishment of versatile human iPSCs capable of gene loading and differentiation into T cells, and disease modeling for aneuploidy syndrome. Thus, engineering of human iPSCs via desired HAC vectors is expected to be widely applied in biomedical research.
| Type: | Article |
|---|---|
| Title: | Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling |
| Location: | United States |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.omtn.2020.12.012 |
| Publisher version: | https://doi.org/10.1016/j.omtn.2020.12.012 |
| Language: | English |
| Additional information: | Copyright © 2020 The Authors. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
| Keywords: | DMD, MV-MMCT, T-iPSC, aneuploidy syndrome, gene and cell therapy, human artificial chromosome |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences > Cell and Developmental Biology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10122079 |
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