Researchers have developed a new technique using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington's disease.
Huntington's is a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. This new approach might block Huntington's from advancing.
Researchers think that the best chance to halt the disease's progression will be to reduce or eliminate the mutant huntingtin (htt) protein found in the neurons of those with the disease.
"For the first time, we have been able to successfully deliver inhibitory RNA sequences from stem cells directly into neurons, significantly decreasing the synthesis of the abnormal huntingtin protein," Jan A Nolta, principal investigator of the study and director of the UC Davis stem cell program and the UC Davis Institute for Regenerative Cures, was quoted as saying.
Huntington's disease can be managed with medications, but currently there are no treatments for the physical, mental and behavioral decline of its victims.
"Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality," Nolta said.
RNA interference (RNAi) technology has been shown to be highly effective at reducing htt protein levels and reversing disease symptoms in mouse models.
"Our challenge with RNA interference technology is to figure out how to deliver it into the human brain in a sustained, safe and effective manner," said Nolta.
"We're exploring how to use human stem cells to create RNAi production factories within the brain," she said.
The findings of this study are available in the article 'Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin' that has been published online in the journal Molecular and Cellular Neuroscience.