Combination treatment in mice shows promise for fatal neurological disorder in kids

Scientists at Washington University have discovered a way to treat Batten disease, an inherited genetic disorder that strikes fewer than five of every 100,000 US children. There are several forms of the disease and all are related to the inability of cells to break down and recycle proteins. In the experiment, improvements were found in life span and motor function of treated mice with gene therapy and bone marrow transplants. Gene therapy was moderately effective in the mice, and bone marrow transplants provided no benefit, but together the two treatments created a striking synergy.The combination therapy did not cure the disease, but mice that received both treatments experienced significant, lasting benefits. Mice that got gene therapy and a bone marrow transplant lived nearly 18.5 months, more than double the lifespan of untreated mice with the disease