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- Gene therapy of Wiskott-Aldrich syndrome: the first successes
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Gene therapy restores immunity in severe congenital pathology
The new method of gene therapy makes it possible to update the immune system of children and young adults with an X-linked form of severe combined immunodeficiency – a hereditary disease that mainly affects male infants.
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Gene therapy: success in the treatment of hereditary disease
Experimental gene therapy has provided a significant improvement in the condition of patients with a rare hereditary disease of the immune system – Wiskott-Aldrich syndrome.
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Hemophilia gene therapy: the first success
After gene therapy, the frequency of spontaneous bleeding in patients with severe hemophilia B decreased by 90%, and the frequency of use of the blood clotting factor IX drug decreased by 92%.
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Gene therapy TKID v.2.0
In the 90s, after genetic therapy of X-linked severe combined immunodeficiency, 25% of patients developed leukemia. This time, a viral vector that does not interact with oncogenes was used.
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Choroideremia gene therapy: success is developing
With the help of gene therapy, scientists from the University of Oxford were able to partially restore vision to six more patients with a hereditary progressive form of blindness – choroideremia.
17 January 2014