Batten disease, a rare and fatal childhood neurological disease, can be treated with
gene therapy to slow the progression of the disease in children. Research conducted at the University of Missuori focused on the treatment of the late infantile form of the disease which is caused by mutations in the gene that encodes for the soluble lysosomal enzyme tripeptidyl peptidase 1, an enzyme that degrades proteins (TPP 1). By injecting dogs with a virus that carries the genes to code for the TPP 1 enzyme into the ventricles, the dogs lived about twice as long. Symptoms of the disease include: vision loss, subtle changes in personality and behavior, slow learning, clumsiness, or stumbling. Dogs that were injected with the treatment showed a delay in developing, or no development whatsoever, symptoms associated with movement, pupil tracking and decision making. When the scientists inspected the dogs brain's they found that the treated dogs had less brain damaged caused by the disease.
Although this treatment has not yet been approved for human trial, this research is promising in offering a treatment to slow the progression of this fatal disease. By using gene therapy, faulty genes are replaced with properly functioning genes, in turn these genes produce functioning proteins which slow the effect of the disease on the brain.
"Dogs that were injected with the treatment showed a delay in developing, or no development whatsoever, symptoms associated with movement, pupil tracking and decision making. When the scientists inspected the dogs brain's they found that the treated dogs had less brain damaged caused by the disease. "This is a incredible way of research. The detection of movement with the treatment can lead to more discoveries on learning about how different animals brains react over time and with growth. Gene therapy can replace bad genes and correct genes that most likely would have decayed.
ReplyDeleteResearch like this advances the medical field tremendously. Because our knowledge of the human genome increases day by day, we are leaning towards a future of healthier people whose illnesses can be cured without the use of painful procedures. The brain still has so many unknown areas, "dark matter", and this shows that humans are delving into that "dark matter" day by day to cure those with sickness.
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