Beta-Thalassemia Clinical Trial
Official title:
The Third Affiliated Hospital of Guangzhou Medical University
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.
1. 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.
2. 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.
3. Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology
system.
4. 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.
5. Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy
and efficiency before the clinical experiment.
6. 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.
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|---|---|---|---|
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