| Description |
On a Good Manufacturing Practice(GMP) condition, we establish non-exotic of different mutation types of beta-thalassemia -induced pluripotent stem cells（iPS） and make a comparisons of the stability and the differentiation of efficiency of these iPS cells inducing from different sources with foreign gene integration.
Using the technique of artificial nuclease and in situ repairmen, we establish efficient system for different beta-thalassemia mutation site and in view of the security of these system.
Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology system.
Build a functional gene therapy self-limiting slow viruses, optimizing the preparation system and to establish a virus preparation of infection of hematopoietic stem cell technology system under the GMP condition.
Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy and efficiency before the clinical experiment.
Improve the existing hematopoietic stem cell transplant(HSCT) clinical application solutions, detect rate of graft rejection, rate of transplantation and other indicators, finish the evaluation of application by clinical cases. |
| Which differentiated cell type is used |
| Label |
hematopoietic stem cell |
| Link |
http://purl.obolibrary.org/obo/CL_0000037 |
| Description |
A stem cell from which all cells of the lymphoid and myeloid lineages develop, including blood cells and cells of the immune system. Hematopoietic stem cells lack cell markers of effector cells (lin-negative). Lin-negative is defined by lacking one or more of the following cell surface markers: CD2, CD3 epsilon, CD4, CD5 ,CD8 alpha chain, CD11b, CD14, CD19, CD20, CD56, ly6G, ter119.; Hematopoietic stem cells (HSCs) are multipotent cells, capable of differentiating into a wide variety of specialized blood cells. During embryonic development hematopoietic stem cells emerge from the hemogenic endothelium, a specialized subset of endothelial cells with hematopoietic potential. HSCs can be found in several organs, in particular in bone marrow, as well as peripheral blood and umbilical cord blood.
These stem cells have a distinctive ability to self-renew, and to differentiate into all lymphoid and myeloid cell types that together form the blood and immune systems including erythrocytes (red blood cells), leukocytes (white blood cells), and platelets. Their primary function is to ensure the continuous replenishment of the blood and immune system throughout an individual's lifespan.
In response to various signals, hematopoietic stem cells can either remain dormant or actively divide and differentiate. Under normal conditions, the majority of these stem cells exist in a quiescent (inactive) state, making them somewhat resistant to chemotherapy or radiation therapy in the treatment of various diseases such as leukemia. However, when the body encounters stress such as infection, bleeding, or disease, hematopoietic stem cells can rapidly become active in order to replenish the affected cells and maintain homeostasis.
Furthermore, hematopoietic stem cells are extremely important in the field of regenerative medicine and transplantation. Regarded as lifesaving in the field of oncology, they are often extracted, purified, and reinfused back into the same (autologous transplantation) or different (allogeneic transplantation) individuals to reestablish a healthy, functioning hematopoietic and immune system after high-dose chemotherapy or to replace dysfunctional bone marrow. Understanding and manipulating the behavior of these stem cells has immense potential in treating various blood disorders, autoimmune diseases, and cancers.
(This extended description was generated by ChatGPT and reviewed by the CellGuide team, who added references, and by the CL editors, who approved it for inclusion in CL. It may contain information that applies only to some subtypes and species, and so should not be considered definitional.); Markers differ between species, and two sets of markers have been described for mice. HSCs are reportedly CD34-positive, CD45-positive, CD48-negative, CD150-positive, CD133-positive, and CD244-negative. |
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