Objective:

To systematically analyze global research trends, conceptual frameworks, and clinical evolution of alternative splicing (AS)-based therapeutics for neurodegenerative diseases (NDDs), highlighting their significance in addressing therapeutic gaps.

Approach:

Key Findings:

• Sustained linear growth in publication output from 2000 to 2025, indicating increasing interest in AS-based therapeutics.
• The United States and Western Europe are dominant collaborative hubs, while China shows high productivity but limited international integration, suggesting potential for enhanced global collaboration.
• LDA topic modeling identified three core conceptual axes: molecular mechanisms, disease-specific pathological models, and translational methodological frameworks, which are crucial for guiding future research.
• Keyword trajectories indicate a shift from in vitro exploration to clinical drug discovery and RNA therapeutics, reflecting the evolving landscape of NDD treatment.
• CLARA clustering revealed a concentration of splicing-modifying interventions in pediatric spinal muscular atrophy (SMA), highlighting the need for similar approaches in adult NDDs.

Interpretation:

The study delineates the structural landscape of AS-based therapeutics for NDDs, indicating a shift from descriptive molecular biology to clinically actionable splicing interventions, which could significantly impact treatment strategies.

Limitations:

• The analysis is limited to publications available in the selected databases (WoSCC and PubMed), which may not encompass all relevant research.
• The study may not capture all relevant research trends outside the specified timeframe, potentially overlooking emerging studies.

Conclusion:

Insights from this study highlight the need to expand targeted RNA platforms beyond SMA into adult neurodegenerative populations, providing a roadmap for future translational research and emphasizing the urgency of addressing adult NDDs.

Attribution Notice

This content is an AI-generated, fully rewritten summary based on a published scholarly article. It does not reproduce the original text and is not a substitute for the original publication. Readers are encouraged to consult the source for full context, data, and methodology.