Funded Studies
Study Rationale:
Previous studies have shown that fat molecules called unsaturated fatty acids regulate the harmful effects that alpha-synuclein have on neurons. This protein forms a key part of Lewy bodies — clumps of proteins, fats and cellular organelles typically found in the brains of people with Parkinson’s disease (PD). Blocking a key enzyme involved in the synthesis of these unsaturated fatty acids, Stearol-CoA-Desaturase 5 (SCD5), could provide a novel treatment for PD that protects brain cells from the toxicity of alpha-synuclein.
Hypothesis:
We hypothesize that inhibiting the production of SCD5 will reduce the accumulation of unsaturated fatty acids and protect brain cells from the damaging effects of alpha-synuclein.
Study Design:
RNA interference is a molecular tool that regulates the process by which a gene directs the synthesis of a protein. This tool is being used to generate a new class of medicines, called RNAi therapeutics (RNAiTh), that can block the production of specific disease-causing proteins. We are designing an RNAiTh that targets the production of SCD5, an approach that should protect human brain cells from the harmful effects of alpha-synuclein in PD. We will test the RNAiTh candidates we generate in multiple PD models and select the most effective molecule to take forward into further studies.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
Studies conducted over the past few years suggest that targeting SCD5 may be protective in slowing the progression of PD.
Next Steps for Development:
The best RNAiTh candidates from this study will be enhanced and tested in additional models to ensure the development of the molecule with the greatest efficacy and fewest side effects for use in clinical trials.