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Abstract
Background: Owing to its biological properties, RNA interference (RNAi) holds the greatest promise as a therapeutic agent for the treatment of a wide range of diseases. The following review is based on the most recent discoveries of RNAi biological mechanisms, highlighting their efficacy, safety, in vivo delivery and use in future clinical trials. Objectives: This review highlights recent advances in RNAi research and focuses on RNAi therapeutic patents for neurodegenerative diseases of the CNS. Method: We used online database free patent resources to summarize RNAi discoveries with particular attention to those regarding the CNS application. We attempted to include the major contributions in the field, and the list of patents reviewed should be considered exhaustive. Conclusion: Patents cover a wide range of RNAi-related fields: molecular findings for understanding RNAi mechanisms, delivery systems and RNAi-based therapeutic applications for neurodegenerative diseases of the CNS.
Acknowledgements
A Orlacchio, G Bernardi, A Orlacchio and S Martino thank the authors cited in this review for their contribution to the development of an alternative therapy using RNAi technology as well as for their work on RNAi molecular mechanisms. They also thank Dr M Renna (CERC-IRCCS Santa Lucia, Rome) for her language advice and Dr F D'Angelo (University of Perugia) for his assistance.