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Updated: Mar 8, 2026

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
Genome-edited safe and immune-evasive human pluripotent cells: Potential solution for allogeneic therapies
Vivian Tam1, Nicole Ching Man Wong1, Andrew Chung Hin Poon1
1School of Biomedical Sciences, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
Abstract:
We used an embryonic stem cell line (H1) engineered for immune-evading properties to avoid rejection ("AlloAccept") and equipped with a "SafeCell" (SC) kill-switch to eliminate aberrantly proliferating cells. Utilizing a humanized immune system mouse model, we demonstrated the successful generation of allogeneic tissues from SafeCell-AlloAccept (SC-AlloAccept) cells in immunocompetent humanized mice in the immune-active subcutaneous region. These cells formed various tissue types, and their growth can be controlled with pro-drug ganciclovir to activate the kill switch, which eliminated proliferating cells and rendered the remaining tissue dormant. Strikingly, SC-AlloAccept-derived grafts survived for 5 months, underscoring their potential for long-term engraftment. Importantly, neither prior rejection of immunogenic parental H1 cells (sensitization) nor the presence of immune-evasive H1-derived tissue (potential immunocompromising) affected the immune response to a subsequent second transplant. This study validated the utility of SC-AlloAccept human cells in transplantation and enhanced the safety and efficacy of stem cell-based regenerative therapies.
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