Scientists have demonstrated the potential for using mesenchymal stem cells (MSCs) to deliver inhibitory RNA sequences targeting the mutant huntingtin gene that is responsible for Huntington disease. Researchers at the University of California Davis Health System’s Institute for Regenerative Cures genetically engineered mesenchymal stem cells derived from the bone marrow of unaffected human donors to carry short hairpin RNAs targeting the huntingtin gene. These MSCs were able to decrease levels of mutant HTT expressed in two different cell lines.
The UC Davis team has been working for the last 20 years to demonstrate that MSCs can be used as safe and effective vehicles for delivering enzymes and proteins to other cells. Reporting their latest work in Molecular and Cellular Neuroscience, Jan A. Nolta, Ph.D., and colleagues say the studies are the first to demonstrate that MSCs can also transfer RNA molecules from one cell to another in amounts sufficient to reduce levels of a mutant protein by over 50%.
"Not only is finding new treatments for Huntington disease a worthwhile pursuit on its own, but the lessons we are learning are applicable to developing new therapies for other genetic disorders that involve excessive protein development and the need to reduce it,” Dr. Nolta remarks. “We have high hopes that these techniques may also be utilized in the fight against some forms of amyotrphic lateral sclerosis as well as Parkinson’s and other conditions.” Their published paper is titled "Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington disease affected neuronal cells for reduction of huntingtin."