Delivering mRNA To Treat Neurological Diseases

By protecting the mRNA in a nanopolymer, scientists have opened up the possibility of using mRNA for gene therapy.

AsianScientist (Mar. 16, 2015) – Rather than use DNA to treat genetic diseases, researchers have developed a method to successfully deliver mRNA instead. This research has been published in The Journal of Controlled Release.

Neurological disorders such as paralysis and Alzheimer’s disease are difficult to cure completely considering the poor capacity of nerve cells to regenerate. Gene therapy is an important treatment strategy because it is possible to correct diseased cells. However, conventional gene therapy techniques such as use of virus vectors and plasmid DNA introduction tend to be difficult in wide clinical applications due to the risk of accidentally inducing mutations in undamaged parts of the genome.

Wild-type messenger RNA (mRNA) is created by transcription from cellular DNA (genes). In this system, instead of administering DNA, artificially created mRNA is introduced into cells to offer a potentially safe and efficient gene therapy. However, mRNA is highly unstable and rapidly degraded in the body, and also strongly stimulates the innate immune system inducing an extremely powerful inflammatory response. As a result, direct mRNA administration into cells has been difficult and there are almost no examples of the application of mRNA as a therapy.

In this study, Associate Professor Keiji Itaka, Professor Kazunori Kataoka and their colleagues in the University of Tokyo Graduate School of Medicine Center for Disease Biology and Integrative Medicine used a polymer micelle drug delivery system to establish a novel mRNA delivery system. Using mRNA for gene therapy, the research group successfully induced tissue regeneration of olfactory nerve fibers and recovery of olfactory function in animals with olfactory disorders. This is the first ever example of successful treatment of a neurological disorder with gene therapy.

These results indicate the feasibility and safety of using mRNA as part of mRNA-based gene therapy and may open the way to applications in many neurological disorders.

The article can be found at: Baba et al. (2015) Treatment of Neurological Disorders by Introducing mRNA in vivo using Polyplex Nanomicelles.


Source: University of Tokyo.
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