Thalassemia Treatment Based on the Stem Cell Technology

General Information

Summary In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.
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.
Clinical trials phase Not applicable
Start date (estimated) 2015-01-01
End date (estimated) 2017-12-31
Clinical feature
Label beta thalassemia
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 NCT03222453
ICTRP weblink
Other study identifiers
Name 201508020258
Source weblink
Public contact
First name Xiaofang
Last name Sun
Phone +86-20-8129 2202
Fax +86-20-8129 2013
Street Duobao road 63
Postcode 510150
City Guangzhou
Sponsors Xiaofang Sun


Which differentiated cell type is used
Label hematopoietic stem cell
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.; 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 Status Unknown Status
Estimated number of participants 2