Breakthrough in a Fatal Childhood Disease
Batten disease is a neurodegenerative disease in children. The disease is caused by a mutation in the TPP1 gene, which is inherited. TPP1 interferes with the brain cell's ability to recycle cellular waste. This leaves the brain with a buildup of waste, which affects the child's ability to walk, talk, think, and see. The average life expectancy for a child with Batten disease is about 10 years. A major breakthrough in research is leading scientists to believe that they have found a way to prolong the affects of the disease, increasing quality of life.
The research was conducted at the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia, and lead by Beverly Davidson. Dogs were used to test out the use of gene therapy. Researchers inserted a working version of the TPP1 gene into dogs. The dogs were not cured of the disease, but their life expectancy increased and it delayed the onset of symptoms. The research does state that it would be beneficial if the outcome was the same for human patients, but there needs to be more work. I look forward to reading more about this research. I am very interested in chronic diseases, and how gene therapy can work to reverse the diseases, or prolong symptoms onset. I do not think that this research is ready to be tried on children quite yet, but I hope to see it develop into a trial soon.