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Scientists say they’ve generated a mouse model that supports the transplantation of human hematopoietic stem cells (HSCs), despite the species barrier and without the need for irradiation.
The group used a mutation of the Kit receptor in the mouse stem cells to facilitate the engraftment of human cells.
In this model, human HSCs can expand and differentiate into all blood cell types without any additional treatment.
Even cells of the innate immune system that are not typically found in “humanized” mice were efficiently generated in this mouse.
Furthermore, the stem cells can be maintained in the mouse over a longer period of time.
The researchers reported these results in Cell Stem Cell.
“Our goal was to develop an optimal model for the transplantation and study of human blood stem cells,” said study author Claudia Waskow, PhD, of Technische Universität Dresden in Germany.
To achieve optimal stem cell engraftment, she and her colleagues introduced a naturally occurring mutation of the Kit receptor into mice lacking a functional immune system.
In this way, the team circumvented the 2 major obstacles of HSC transplantation: the rejection by the recipient’s immune system and the absence of free niche space for the incoming donor stem cells in the recipient’s bone marrow.
The Kit mutation in the new mouse model impairs the recipient’s stem cell compartment in such a way that the endogenous HSCs can be easily replaced by human donor stem cells with a functional Kit receptor.
The researchers said this replacement works so efficiently that irradiation can be completely omitted, allowing the study of human blood development in a physiological setting. The model can now be used to study diseases of the human blood and immune system or to test new treatment options.
The results of this research also show that the Kit receptor is important for the function of human HSCs, notably in a transplant setting. The researchers said future studies will focus on using this knowledge to improve conditioning therapy for patients undergoing HSC transplant. 
Scientists say they’ve generated a mouse model that supports the transplantation of human hematopoietic stem cells (HSCs), despite the species barrier and without the need for irradiation.
The group used a mutation of the Kit receptor in the mouse stem cells to facilitate the engraftment of human cells.
In this model, human HSCs can expand and differentiate into all blood cell types without any additional treatment.
Even cells of the innate immune system that are not typically found in “humanized” mice were efficiently generated in this mouse.
Furthermore, the stem cells can be maintained in the mouse over a longer period of time.
The researchers reported these results in Cell Stem Cell.
“Our goal was to develop an optimal model for the transplantation and study of human blood stem cells,” said study author Claudia Waskow, PhD, of Technische Universität Dresden in Germany.
To achieve optimal stem cell engraftment, she and her colleagues introduced a naturally occurring mutation of the Kit receptor into mice lacking a functional immune system.
In this way, the team circumvented the 2 major obstacles of HSC transplantation: the rejection by the recipient’s immune system and the absence of free niche space for the incoming donor stem cells in the recipient’s bone marrow.
The Kit mutation in the new mouse model impairs the recipient’s stem cell compartment in such a way that the endogenous HSCs can be easily replaced by human donor stem cells with a functional Kit receptor.
The researchers said this replacement works so efficiently that irradiation can be completely omitted, allowing the study of human blood development in a physiological setting. The model can now be used to study diseases of the human blood and immune system or to test new treatment options.
The results of this research also show that the Kit receptor is important for the function of human HSCs, notably in a transplant setting. The researchers said future studies will focus on using this knowledge to improve conditioning therapy for patients undergoing HSC transplant.
Scientists say they’ve generated a mouse model that supports the transplantation of human hematopoietic stem cells (HSCs), despite the species barrier and without the need for irradiation.
The group used a mutation of the Kit receptor in the mouse stem cells to facilitate the engraftment of human cells.
In this model, human HSCs can expand and differentiate into all blood cell types without any additional treatment.
Even cells of the innate immune system that are not typically found in “humanized” mice were efficiently generated in this mouse.
Furthermore, the stem cells can be maintained in the mouse over a longer period of time.
The researchers reported these results in Cell Stem Cell.
“Our goal was to develop an optimal model for the transplantation and study of human blood stem cells,” said study author Claudia Waskow, PhD, of Technische Universität Dresden in Germany.
To achieve optimal stem cell engraftment, she and her colleagues introduced a naturally occurring mutation of the Kit receptor into mice lacking a functional immune system.
In this way, the team circumvented the 2 major obstacles of HSC transplantation: the rejection by the recipient’s immune system and the absence of free niche space for the incoming donor stem cells in the recipient’s bone marrow.
The Kit mutation in the new mouse model impairs the recipient’s stem cell compartment in such a way that the endogenous HSCs can be easily replaced by human donor stem cells with a functional Kit receptor.
The researchers said this replacement works so efficiently that irradiation can be completely omitted, allowing the study of human blood development in a physiological setting. The model can now be used to study diseases of the human blood and immune system or to test new treatment options.
The results of this research also show that the Kit receptor is important for the function of human HSCs, notably in a transplant setting. The researchers said future studies will focus on using this knowledge to improve conditioning therapy for patients undergoing HSC transplant.