Evidence supporting the use of: RNA
For the health condition: Parkinson's Disease

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Synopsis

Source of validity: Scientific
Rating (out of 5): 2

RNA-based therapies have garnered scientific interest for the treatment of Parkinson’s Disease (PD), but their use remains experimental and is not yet a standard of care. The rationale stems from the molecular mechanisms underlying PD, which involve the misfolding and aggregation of alpha-synuclein protein and the degeneration of dopaminergic neurons. Some RNA-based approaches under investigation include antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and messenger RNA (mRNA) therapies. These strategies aim to reduce the production of pathogenic proteins or to modulate gene expression implicated in PD pathogenesis. For example, preclinical studies in animal models have demonstrated that ASOs targeting alpha-synuclein mRNA can reduce alpha-synuclein levels and ameliorate motor symptoms. Additionally, several early-phase clinical trials are ongoing to evaluate the safety and efficacy of RNA-based interventions in human PD patients.

Despite promising data from laboratory and animal research, there is currently insufficient high-quality clinical evidence to fully endorse RNA therapies for PD in humans. No RNA-based therapy has received regulatory approval for this indication as of June 2024, and most data come from preclinical or early-phase clinical studies. Therefore, while there is a scientific rationale and some early experimental support, the evidence base is still developing, and these treatments are not yet widely available or validated for routine clinical use in Parkinson’s Disease.

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