Researchers from the University of Kentucky presented the results of their research on a potential novel therapy for the treatment of Parkinson’s disease. The poster presentation, entitled “Therapeutic Development of siRNA targeting Alpha-Synuclein,” was part of the Michael J. Fox Foundation (MJFF) Parkinson’s Disease Therapeutics Conference held in New York on Nov. 3. It is only conference specifically discussing the development of Parkinson’s disease therapies.
The study, led by Professor Greg Gerhardt and Associate Professor Richard Grondin, both from the university’s College of Medicine, together with a pharmaceutical company and the MJFF, aims to understand if targeting the protein α-synuclein is a safe therapy approach to treat Parkinson’s disease (PD). This protein has become central in PD research due to its potential role in both familial and sporadic disease pathogenesis and progression. This presynaptic neuronal protein is present in excess in Lewy bodies, neuron-damaging structures made of waste proteins that neurons fail to recycle. The aggregation mechanism of α-synuclein along with the protein’s function in the brain are still being investigated. Recent research focuses on therapies targeting this protein but also on its validity as a PD biomarker.
Dr Gerhardt’s research team is testing a siRNA (small interfering RNA) approach targeting α-synuclein mRNA which is able to stop its expression. siRNA are double-stranded synthetic RNA molecules that complement specific mRNA sequences, causing its breakdown and stopping protein translation. This method has been widely used in basic biomedical research and also in drug development. In vivo results have shown it effective to stop α-synuclein formation without toxic effects on the brain, and as such it seems that siRNA can become a promising therapeutic candidate for the treatment of Parkinson’s disease.
By reducing α-synuclein gene expression or blocking its aggression to neurons, researchers hope to develop an effective therapy that will target the disease before irreversible damage occurs, rather than just manage disease symptoms.
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