Wednesday, 22 April 2015

Groundbreaking - A Cure For Genetic Disorders

Daniel Wheeler, 15, has been 'miraculously' cured of Wiskott-Aldrich Syndrome, a rare genetic blood condition. He was diagnosed with the condition aged two and had needed frequent medical care to manage his symptoms which included severe eczema, asthma and inability to fight infections. His older brother died from complications linked to the disease.

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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