A recent clinical trial conducted at Great Ormond Street Hospital in London, England, has successfully treated seven patients suffering from the genetic disorder Wiskott-Aldrich Syndrome by utilizing autologous [the patient’s own] stem cells. The therapy was an example of translational genomics, in which doctors extract autologous stem cells, correct the faulty gene that causes Wiskott-Aldrich Syndrome, and then implant the stem cells back into the patients to produce new, healthy cells.
Recently, researchers utilized stem cells to successfully treat six children with rare genetic diseases. Three of the children were born with Metachromatic Leukodystrophy (MLD), a hereditary neurodegenerative disease. The other three children were born with Wiskott-Aldrich Syndrome (WAS), a hereditary immune system disorder.