Understanding a Devastating Childhood Disease is Now One Step Closer
New research from the National Cancer Institute provides hope for children with "Hutchinson-Gilford progeria syndrome" — a rare and fatal disease that causes the body to age at an extreme rate.
Akinso: It's a disease that affects one out of eight-million infants — and causes them to grow old before their time: a rare and fatal disease called Hutchinson-Gilford Progeria Syndrome causes the body to age at an extreme rate. But, now, there's hope that cells affected by this disease can be made healthy again — according to a study conducted by researchers at the National Cancer Institute. One of the researchers — Doctor Tom Misteli — believes this study could bring scientists one step closer to understanding this devastating childhood disease.
Mistelli: What's amazing to me is that you can take a defective cell from a patient, and you can eliminate this one bad protein, and the cell still has the potential to function perfectly normally. So, the cell really is able to recover from this defect. So, if we can find a way to eliminate this mutant protein in patient cells, there's no reason why these cells shouldn't behave normally. That obviously would be a very significant step toward therapy.
Akinso: The symptoms of Hutchinson-Gilford Progeria Syndrome are stunted growth, loss of hair and body fat, joint stiffness, osteoporosis, and heart problems — which, according to Doctor Misteli, constitutes the leading cause of death. Affected children usually die before they reach their late teens. This is Wally Akinso, at the National Institutes of Health, Bethesda, Maryland.
About This Audio Report
Reporter: Wally Akinso
Sound Bite: Dr. Tom Mistelli