US researchers have developed an innovative technique that uses stem cells to deliver an RNA interference (RNAi) based therapeutic for Huntington’s disease.
Huntington’s disease (HD) is an inherited form of adult-onset, severe progressive neurological condition that results in deteriorating physical and mental abilities. There is no effective treatment to prevent disease progression. Now, publishing in Molecular and Cellular Neuroscience, scientists report a new approach that could help slow or even halt disease progression.
RNA interference has previously been shown to be effective at reducing levels of the abnormal huntingtin (htt) protein that cause disease in mouse models; however, delivery of RNA therapies to the human brain is a difficult problem. The new technique used inhibitory RNA sequences inserted into stem cells from healthy donors; the resulting stem cells were injected to recipient nerve cells, with a resulting decrease in levels of the disease-linked htt protein.
Lead researcher Jan A. Nolta, of the UC Davis Institute for Regenerative Cures said: "Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality".
Comment:This research is at a fairly early stage, requiring further work, and also represents an expensive and invasive form of therapy. However, given the severity of HD and the current lack of effective treatments, it could indeed signal important progress for affected patients and families.