New Hope for Huntington’s disease

A story posted in the Medical News Today Janurary 14, 2012, states that new hope has been discovered for Huntington’s disease. Last year a research study conducted by Jan A. Nolta (UC Davis stem cell program and UC Davis Institute for Regenerative Cures) was published in the journal Molecular and Cellular Neuroscience that they may be able to provide gene therapy for individuals affected with Huntington's Disease by using inhibitory RNA sequences to reduce the amount of the mutant huntingtin protein. Huntington’s disease is a neurological degenerative genetic disorder that is caused by the mutation of the huntingtin gene, comprised of the abnormal repeating of the building block of DNA that appears on the fourth chromosome. The accumulation of the huntingtin protein in the brain of an individual leads to progressive uncontrolled movements, dementia and death.

In the study conducted by using a mouse model, the research team genetically created mesenchymal stem cells that were made from the bone marrow of the individuals that were unaffected with the disease to transfer the inhibitory RNA sequences from donor cells to the target cells. From this transfer, the amount of the huntingin protein present dropped significantly by over 50 %. The research team has hopes of transferring the RNA inference technology over to humans in a “sustained, safe and effective manner”.

It is intriguing to find that researchers have found a way to reduce the Huntingtin protein and even possibly eliminating the disease all together.  Huntington’s disease usually does not appear in individuals until they reach middle age, so individuals who have not developed the signs and symptoms the chance to stop the progression of the disease for it begins; also help the individuals already suffering from the disease before it progresses further.