New hope for patients with Mediterranean anemia

The report of an international team of scientists showed that it was initially successful in gene therapy for a patient with Mediterranean anemia.

This patient suffers from Hải Mediterranean anemia, and at the age of three had to undergo blood transfusions, the period of severe illness each month must be transfused once. Three years ago, at the age of 18, this patient began receiving gene therapy.

Mediterranean anemia is caused by a deficiency of the gene , the patient cannot produce normal red blood cells, so blood transfusions are needed to maintain the nourishing function of the blood.

In the course of gene therapy experiments, scientists first cultured blood cells including red blood cells, by taking advantage of hematopoietic stem cells in the patient's marrow. Then use the virus as a transmitter to put the complete gene into the blood cells and continue using chemical methods to remove excess cells, leaving only red blood cells. Finally, transplanting red blood cells into the patient's body.

The results show that the patient's ability to produce normal red blood cells has gradually increased.After one year of receiving gene therapy, the patient did not need blood transfusions .

The initial success of the experiment opened up a great prospect for gene therapy for Mediterranean anemia, and hoped for many patients with blood diseases.