LOS ANGELES, April 21 (UPI) -- University of Southern California scientists say they've made a genetic discovery that might lead to gene therapy treatments for Huntington's disease.
Huntington's disease is an incurable neurological disorder characterized by uncontrolled movements, emotional instability and loss of intellectual faculties.
"Our findings allow for the possibility that controlled over-expression of RCAN1-1L might in the future be a viable avenue for therapeutic intervention in Huntington disease patients," said Professor Kelvin Davies, who led the study.
Davies and his colleagues used cell-culture findings to show a form of the gene RCAN1, known as RCAN1-1L, is dramatically decreased in human brains affected by Huntington's disease. The researchers also showed increasing levels of RCAN1-1L rescues cells from the toxic effects of Huntington's disease, a result Davies says might lead to new avenues of treatment.
"It is important to keep in mind that these protective findings are in-vitro, meaning in cell cultures," said Associate Professor Gennady Ermak, lead author of the research. "Further proof of protection by RCAN1-1L will be required in-vivo, or in actual Huntington disease patients."
The study that will appear in the June issue of the Journal of Biological Chemistry is now available online at the journal's Web site.
