Wednesday, 22 April 2015
Groundbreaking - A Cure For Genetic Disorders
Daniel partook in clinical trials for a new gene therapy in 2011, alongside 7 other children. Since then, he has been symptom free and doctors say he has been effectively cured.
Wiskott-Aldrich syndrome is characterized by abnormal immune system function (immune deficiency) and a reduced ability to form blood clots. This condition primarily affects males.
Individuals with Wiskott-Aldrich syndrome have microthrombocytopenia, which is a decrease in the number and size of blood cells involved in clotting (platelets). This platelet abnormality, which is typically present from birth, can lead to easy bruising or episodes of prolonged bleeding following minor trauma.
The research was published in the Journal of the American Medical Association.
This offers hope that other genetic conditions with time, may become curable.
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