Researchers may have found a way to silence the gene for Huntington’s disease. In animals, scientists were able to slow the progression of the disease, and even reverse its effects, for up to nine months.
Huntington’s disease sufferers, as well as their family members and caretakers, have long been unable to find solace in medicine. A degenerative cognitive and physical disease, Huntington’s disease affects 30,000 people in the United States, with symptoms that include uncontrolled movements and progressive cognitive and psychological problems.
Huntington's disease is caused by the mutation of a single gene, chromosome 4, which causes a part of the DNA to repeat many more times than it is supposed to. In a healthy person, the CAG, as it is called, is repeated 10 to 28 times. In sufferers from Huntington’s disease, it is repeated 36 to 120 times. It is transmitted by a parent, and affects both adults and children. As of yet there is no effective treatment for curing or even delaying the disease.
Led by Don W. Cleveland, PhD, professor and chair of the University of California San Diego Department of Cellular and Molecular Medicine and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research; the team injected mouse and non-human primate models of Huntington’s disease with a one-time shot of a DNA drug based on antisense oligonucleotides (ASOs). These ASO's selectively attach to and eliminate the mutated gene’s molecular instructions for creating the huntingtin protein.
The team discovered that the injection helped the animals for up to nine months with motor skills and cognitive problems. In mouse models, the injection even reversed brain atrophy and increased the lifespans of the mice in which Huntington's disease had already considerably progressed. Surprisingly, the injection’s effects remained in place for up to nine months, even after the actual ASO was no longer in the animal’s system.
Researchers are optimistic for future use of the drug. TThis is merely the first step in a lengthy process, researchers spoke of a "Huntington’s holiday," wherein suffers could spend months without symptoms of their debilitating condition.
The findings are printed in a recent issue of Neuron.