huHSC-NCG-X

Proto-oncogene c-KIT, also known as CD117 (cluster of differentiation 117 or mast/ stem cell growth factor receptor (SCFR)), is a receptor tyrosine kinase protein that is encoded by the KIT gene. Generally, hematopoietic stem/progenitor cells express c-kit, while acute lymphoblastic leukemia cells rarely express it. The c-KIT protein plays a crucial function in the signaling pathway for bone marrow-to-plasma migration of hematopoietic progenitor cells. 

C-KIT mutations have been linked to multiple types of cancer. The W41 is a point mutation at position 831 Val to Met in the c-kit protein, which may  suppress the function of hematopoietic stem cells, resulting in spontaneous anemia in mice.

GemPharmatech developed the NCG-kit-cas9-TM (NCG-X) strain, which does not require irradiation during HSC reconstitution, and huHSC-NCG-X can reconstitute red lineage cells in mouse bone marrow that could be utilized to research thalassemia.


Applications

  • Human immune system development

  • Research on thalassemia

  • Evaluation of anti-tumor drugs targeting red blood cells

  • Immune system reconstitution models that do not require irradiation or chemoablation

 

Supporting data

1. Human immune reconstitution levels of huHSC-NCG-X mice

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Compared with irradiated NCG mice, huHSC-NCG-X mice were able to rebuild the human immune system efficiently without irradiation.


2. Reconstitution levels of red blood cells in huHSC-NCG-X mice in the bone marrow

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The human HSC reconstruction assays in NCG-X mice demonstrated a considerable increase in the proportion of reconstructed erythrocytes in the bone marrow of NCG-X mice compared to irradiated NCG mice. This finding suggests that huHSC-NCG-X can be used as a good model to research diseases like thalassemia.


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