iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

General Information

Summary This is a single centre, single arm, open-label study, to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
Description The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
Clinical trials phase Other
Start date (estimated) 2019-01-01
End date (estimated) 2021-01-31
Clinical feature
Label beta thalassemia
Link http://purl.obolibrary.org/obo/DOID_12241
Description A thalassemia characterized by the reduced or absent synthesis of the beta globin chains of hemoglobin.; Xref MGI. OMIM mapping confirmed by DO. [SN].

Administrative Information

NCT number NCT03728322
ICTRP weblink https://trialsearch.who.int/Trial2.aspx?TrialID=NCT03728322
Other study identifiers
Name HBB HSC-01
Source weblink https://clinicaltrials.gov/ct2/show/NCT03728322
Sponsors Allife Medical Science and Technology Co., Ltd.

Cells

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.

Recruitment

Recruitment Status Not yet recruiting
Estimated number of participants 12