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.
Scientists led by Mathias Hoehn at the Max Planck Institute for Metabolic Research have devised a novel method for documenting the process by which human stem cells transplanted into the cerebral cortex of an animal model develop into mature nerve cells. The researchers inserted optical image reporters into the cells to emit different shades of light when certain genes were activated. By observing the sequence in which the light was emitted, the group determined the timeline of the stem cell’s transformation in vivo.