Illustration of Gene Therapy Approach for Pompe Disease
This is an illustration of this gene therapy approach for Pompe disease, a deadly, inherited muscle disorder caused by a faulty gene. This image depicts a modified virus, called rAAV 8, being injected into an individual and carrying a healthy gene to the liver. Once in the liver, the gene produces a missing enzyme, called GAA, which goes to the muscles and helps them work. Based on mouse studies, the researchers also expect the gene therapy to train the body’s immune system to recognize GAA and not attack it.
Credit: National Center for Advancing Translational Sciences
Illustration of Gene Therapy Approach for Pompe Disease
This is an illustration of this gene therapy approach for Pompe disease, a deadly, inherited muscle disorder caused by a faulty gene. This image depicts a modified virus, called rAAV 8, being injected into an individual and carrying a healthy gene to the liver. Once in the liver, the gene produces a missing enzyme, called GAA, which goes to the muscles and helps them work. Based on mouse studies, the researchers also expect the gene therapy to train the body’s immune system to recognize GAA and not attack it.
Credit: National Center for Advancing Translational Sciences