Pharmaceutical Research

, Volume 23, Issue 8, pp 1631–1640

Ligand-Targeted Delivery of Therapeutic siRNA

Authors

  • Yutaka Ikeda
    • Gene Function Research CenterNational Institute of Advanced Industrial Science and Technology (AIST)
    • Gene Function Research CenterNational Institute of Advanced Industrial Science and Technology (AIST)
    • Department of Chemistry and Biotechnology, School of EngineeringThe University of Tokyo
Expert Review

DOI: 10.1007/s11095-006-9001-x

Cite this article as:
Ikeda, Y. & Taira, K. Pharm Res (2006) 23: 1631. doi:10.1007/s11095-006-9001-x

Abstract

RNA interference (RNAi) is a post-transcriptional gene-silencing phenomenon that is triggered by double-stranded RNA (dsRNA). Since many diseases are associated with the inappropriate production of specific proteins, attempts are being made to exploit RNAi in a clinical settings. However, before RNAi can be exploited as therapeutically, several obstacles must be overcome. For example, small interfering RNA (siRNA) is unstable in the blood stream so any effects of injected siRNA are only transient. Accordingly, methods must be developed to prolong its activity. Furthermore, the efficient and safe delivery of siRNA into target tissues and cells is critical for successful therapy. Any useful delivery method should be designed to target siRNA to specific cells and to promote gene-silencing activity once the siRNA is inside the cells. Recent chemical modifications of siRNA have overcome problems associated with the instability of siRNA, and various ligands, including glycosylated molecules, peptides, proteins, antibodies and engineered antibody fragments, appear to be very useful or have considerable potential for the targeted delivery of siRNA. The use of such ligands improves the efficiency, specificity and, as a consequence, the safety of the corresponding delivery systems.

Key words

antibodyantibody engineeringligandRNA interferencetargeted delivery

Copyright information

© Springer Science + Business Media, Inc. 2006