RNA-Based Therapies for Neurodegenerative Diseases Targeting Pathogenic Proteins

Area of Science:

• Neuroscience
• Molecular Biology
• Genetics

Background:

• Neurodegenerative diseases (NDDs) are characterized by progressive neuronal dysfunction and loss, leading to severe motor and cognitive deficits.
• Key pathological hallmarks include protein aggregate accumulation, neuroinflammation, and neuronal apoptosis.
• Alzheimer's disease (AD) and Parkinson's disease (PD) exemplify NDDs, affecting over 65 million individuals worldwide with increasing prevalence.

Purpose of the Study:

• To review the pathogenic roles of protein aggregates in NDDs.
• To highlight emerging RNA-based therapeutic strategies for targeting these pathological proteins.
• To underscore the need for novel treatments beyond symptom management.

Main Methods:

• Literature review of studies on NDD pathogenesis.
• Analysis of research on protein aggregation in Alzheimer's and Parkinson's diseases.
• Exploration of current and emerging RNA-based therapeutic modalities.

Main Results:

• Protein aggregates are central to NDD pathology, driving neuronal dysfunction and death.
• Existing treatments primarily manage symptoms and have side effects.
• RNA therapeutics present a novel molecular intervention strategy for NDDs.

Conclusions:

• Targeting pathological protein aggregates is crucial for developing effective NDD therapies.
• RNA-based therapeutics show significant potential for halting or slowing neurodegeneration.
• Further research into RNA-based strategies is essential for advancing NDD treatment options.