Gene Therapy Helps Children in German Study

Wiskott-Aldrich-syndromeResearchers in Germany were able to use gene therapy to correct a rare blood disorder which affects children to devastating results.

Scientists ‘fixed’ a malfunctioning gene which is the cause of Wiskott-Aldrich syndrome, a rarely seen but highly serious childhood illness that causes prolonged bleeding from even the mildest of cuts. This disease also leaves children extremely vulnerable to dangerous infections and some cancers.

“The study shows proof-of-principle that gene therapy with stem cells in a genetic disorder like this has strong potential,” added Paul Sanberg, a stem cell specialist who is director of the University of South Florida Center of Excellence for Aging and Brain Repair in Tampa.