Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling
| Autor: | Shin Kaneko, Mitsuo Oshimura, Masaki Sugawara, Giulia Ferrari, Kanako Kazuki, Kazuma Tomizuka, Yasuhiro Kazuki, Satoshi Nishikawa, Satoshi Abe, Chiaki Sawada, Atsushi Kunisato, Akane Okada, Naoyo Kajitani, Yuwna Yakura, Ken Fukumoto, Mitsuhiko Osaki, Shinichiro Takayanagi, Narumi Uno, Shuta Takata, Masaharu Hiratsuka, Francesco Tedesco, Yuichi Nagashima |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
0301 basic medicine
human artificial chromosome gene and cell therapy Chromosome Transfer Computational biology Human artificial chromosome Biology Cell therapy 03 medical and health sciences 0302 clinical medicine Plasmid DMD Drug Discovery Vector (molecular biology) Induced pluripotent stem cell Gene T-iPSC Drug discovery lcsh:RM1-950 aneuploidy syndrome lcsh:Therapeutics. Pharmacology 030104 developmental biology 030220 oncology & carcinogenesis Molecular Medicine Original Article MV-MMCT |
| Zdroj: | Molecular Therapy-Nucleic Acids Molecular Therapy. Nucleic Acids Molecular Therapy: Nucleic Acids, Vol 23, Iss, Pp 629-639 (2021) |
| ISSN: | 2162-2531 |
| Popis: | 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. Graphical Abstract Engineering of human iPSCs has great potential for cell therapy and drug discovery. Kazuki and colleagues demonstrate engineering of human iPSCs via human artificial chromosome vectors with a large cargo capacity for biomedical research, such as gene and cell therapies and generation of isogenic models for aneuploid syndromes. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|