Due to changes in genetic information, the patient's cells began to produce the children's form of hemoglobin instead of the adult one.
CRISPR Therapeutics Company has for the first time treated patients with beta thalassemia and sickle cell anemia, which are genetic inherited diseases. By correction the blood cell genome, the company's specialists have managed to partially cope with these conditions.
After the therapy, one of the patients with beta thalassemia can completely discontinue blood transfusions. Another woman with sickle cell anemia no longer suffers from the vascular blockage characteristic of this disease.
The CRISPR technology has enabled the doctors to edit the cell genome making them produce the child form of hemoglobin instead of an adult one.
CRISPR Therapeutics plans to develop methods to treat malignant blood cell diseases, such as various types of leukemia.
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