RNA interference as an anticancer therapy: a patent perspective.
BACKGROUND
The development of RNA interference (RNAi)-based gene silencing approaches has revolutionized biomedical research. These technologies have been applied for functional genomic studies in a variety of areas, including cancer research, by facilitating a better understanding of the mechanisms that underlie tumorigenicity and the identification of novel factors that either promote or inhibit oncogenic transformation.


OBJECTIVE
These approaches have laid the groundwork for the development of a novel class of genetic therapies. Preclinical results have exposed both the unique therapeutic opportunities and challenges that are encountered in adapting these technologies for clinical applications. These themes are reflected in the patent literature that has mirrored the rise in complexity and sophistication of RNAi-based therapeutic approaches. This review focuses on the identification of potential anticancer therapeutic targets and the development of clinically relevant delivery approaches.


CONCLUSIONS
Thus far, the patent landscape in the RNAi field has been dominated by a handful of key patents that describe the original identification and characterization of inhibitory double-stranded RNA molecules. Only time will tell how these original patents will hold up in the face of the development of new approaches and reagents as RNAi-based therapeutics approach transition from the bench to the clinic.
The University of Miami Miller School of Medicine, Miami Institute for Human Genomics, The John T Macdonald Foundation, Department of Human Genetics, Miami, FL 33136, USA. DDykxhoorn@med.miami.edu
Derek M
DM
Dykxhoorn
D000818:Animals; D020868:Gene Silencing; D018390:Gene Targeting / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D009369:Neoplasms / Q000628:therapy*; D010330:Patents as Topic; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000627:therapeutic use
D034741:RNA, Small Interfering