Genetic engineering of immune evasive stem cell-derived islets Review


Authors: Sackett, S. D.; Kaplan, S. J.; Mitchell, S. A.; Brown, M. E.; Burrack, A. L.; Grey, S.; Huangfu, D.; Odorico, J.
Review Title: Genetic engineering of immune evasive stem cell-derived islets
Abstract: Genome editing has the potential to revolutionize many investigative and therapeutic strategies in biology and medicine. In the field of regenerative medicine, one of the leading applications of genome engineering technology is the generation of immune evasive pluripotent stem cell-derived somatic cells for transplantation. In particular, as more functional and therapeutically relevant human pluripotent stem cell-derived islets (SCDI) are produced in many labs and studied in clinical trials, there is keen interest in studying the immunogenicity of these cells and modulating allogeneic and autoimmune immune responses for therapeutic benefit. Significant experimental work has already suggested that elimination of Human Leukocytes Antigen (HLA) expression and overexpression of immunomodulatory genes can impact survival of a variety of pluripotent stem cell-derived somatic cell types. Limited work published to date focuses on stem cell-derived islets and work in a number of labs is ongoing. Rapid progress is occurring in the genome editing of human pluripotent stem cells and their progeny focused on evading destruction by the immune system in transplantation models, and while much research is still needed, there is no doubt the combined technologies of genome editing and stem cell therapy will profoundly impact transplantation medicine in the future. Copyright © 2022 Sackett, Kaplan, Mitchell, Brown, Burrack, Grey, Huangfu and Odorico.
Keywords: stem cell transplantation; genetic engineering; pluripotent stem cell; pluripotent stem cells; regenerative medicine; pancreas islet; islets of langerhans; humans; human; crispr; gene editing; allograft rejection; hla allobarrier; type i diabetes
Journal Title: Transplant International
Volume: 35
ISSN: 0934-0874
Publisher: Wiley Blackwell  
Date Published: 2022-01-01
Start Page: 10817
Language: English
DOI: 10.3389/ti.2022.10817
PUBMED: 36545154
PROVIDER: scopus
PMCID: PMC9762357
DOI/URL:
Notes: Review -- Export Date: 3 January 2023 -- Source: Scopus
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  1. Danwei Huangfu
    56 Huangfu
  2. Samuel Joseph Kaplan
    4 Kaplan