Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling.
DMD
MV-MMCT
T-iPSC
aneuploidy syndrome
gene and cell therapy
human artificial chromosome
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
05 Mar 2021
05 Mar 2021
Historique:
received:
29
06
2020
accepted:
11
12
2020
entrez:
8
2
2021
pubmed:
9
2
2021
medline:
9
2
2021
Statut:
epublish
Résumé
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.
Identifiants
pubmed: 33552683
doi: 10.1016/j.omtn.2020.12.012
pii: S2162-2531(20)30393-0
pmc: PMC7819819
doi:
Types de publication
Journal Article
Langues
eng
Pagination
629-639Subventions
Organisme : Medical Research Council
ID : MR/J006785/1
Pays : United Kingdom
Informations de copyright
© 2020 The Authors.
Déclaration de conflit d'intérêts
K.F., S. Takayanagi, and A.K. are employed by Kirin Holdings, Co., Ltd. S.N. is employed by Kyowa Kirin, Co., Ltd. The other authors declare no competing interests.
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