Gene therapy of Wiskott-Aldrich syndrome: the first successes
Scientists: gene therapy will help in the fight against the deadly syndrome
Scientists have received the first positive results of the use of gene therapy in the treatment of Wiskott-Aldrich syndrome, this was announced at the annual meeting of the American Society of Hematology.
Wiskott-Aldrich syndrome is a rare X–linked recessive disease characterized by the presence of eczema, thrombocytopenia, immunodeficiency and bloody diarrhea. Exclusively male persons suffer from the syndrome. The prognosis of treatment is usually unfavorable, but with bone marrow transplantation, the chances of boys living to the age of 30 increase significantly.
According to the data presented at the meeting, four boys suffering from Wiskott-Aldrich syndrome underwent gene therapy, and after treatment they had a significant improvement in their health. The authors of the study believe that further extensive and long-term studies are needed in order to obtain the most complete data on the effectiveness of gene therapy in the fight against the syndrome.
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08.12.2015