地中海贫血_BDgene

Thalassemia

Lentiviral vector transduce autologous CD34+ hematopoietic stem cells to treat transfusion-dependent β-thalassemia, and 2 case of IIT human clinical has been completed. This is the first successful case of treating thalassemia major based on lentiviral vector gene transduction technology in China.

Thalassemia

Thalassemia is a hereditary hemolytic anemia caused by the globin production disorder caused by the mutation or deletion of the hemoglobin globin gene. It is a typical single-gene genetic disease, mainly including α-thalassemia and β-thalassemia .

According to the "China Blue Book of Thalassemia Prevention and Treatment (2015)", there are currently about 30 million "thalassaemia" gene carriers in my country, of which about 300,000 patients with severe and intermediate thalassaemia are at a rate of about 10% per year. Increment.

Gene Therapy Of Thalassemia

The "thalassaemia" gene therapy developed by BDgene is promising: "One treatment, life-long cure". Firstly, the patient’s hematopoietic stem cells are isolated, activated and cultured in vitro, the correct β-globin gene is compensated to the patient’s hematopoietic stem cells through BDgene's unique lentiviral vector, and then a series of functional and safety tests are performed in vitro , And then return the hematopoietic stem cells with the correct gene to the patient for hematopoietic stem cell transplantation to re-establish and repair the patient’s hematopoietic system.

Thalassemia(图1)