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项与 ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia) 相关的临床试验Phase 1/2 Study Evaluating the Safety and Efficacy of Gene Therapy Employing Lentiviral Vector ALS20-transduced Hematopoietic Progenitor Cells in Subjects With Transfusion-dependent-thalassemia
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.
100 项与 ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia) 相关的临床结果
100 项与 ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia) 相关的转化医学
100 项与 ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia) 相关的专利(医药)
100 项与 ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia) 相关的药物交易