Klein working with five-year old Felix Ott, who was diagnosed with Wiskott-Aldrich Syndrome at age three. When he was four, Felix received stem-cell gene therapy, and the now seven-year-old has since been able to live a normal life.VERENA MULLER
The two 3-year-olds were very, very sick. One was bleeding internally, suffered from severe eczema and anemia, and had multiple infections in his lungs and colon. The other had a dangerously low platelet count, recurring respiratory tract infections, and a life-threatening salmonella infection in his blood. Both turned up in pediatric hematologist Christoph Klein’s office in 2006, their parents clutching tightly to the hope that Klein might be able to save their children’s lives.
Klein, then at the Hannover Medical School in Germany, was running the first-ever gene therapy trial for Wiskott-Aldrich Syndrome, a rare and life-threatening disease caused by mutations in WAS, a gene whose protein product normally regulates the formation of actin polymers in hematopoietic cells. A dearth of functional WAS protein causes ...
