International
US researchers have used gene therapy to treat eight toddlers with a genetic disorder known as Hurler syndrome, which can be fatal without treatment. Kids with the condition lack an enzyme called α-L-iduronidase (IDUA) and the condition is one of a group of disorders known as of lysosomal storage diseases. It can be treated by stem cell transplant from a donor, but matched donors are not always available and the disease can still progress after treatment, severely affecting patients’ quality of life. To overcome these challenges the researchers used the patients own stem cells and genetically engineered them to overproduce the missing enzyme. Around 2 years after the procedure, all patients were still making their own enzyme, the safety profile was similar to donor stem cell transplant and there were encouraging early clinical outcomes.
Note: Not all attachments are visible to the general public. Research URLs will go live after the embargo ends.
Sign In
