BACKGROUND: RNA interference (RNAi) is a powerful endogenous process initiated by short double-stranded RNAs, which results in sequence-specific posttranscriptional gene silencing. The possibility of blocking the expression of any protein carries huge expectations for potential therapeutic applications in a wide range of diseases. For clinical development, however, the use of RNAi-based therapeutics has to overcome major obstacles, mainly targeted delivery and safety issues. OBJECTIVE/ METHODS: In this short review, we provide an overview of specifications for RNAi-based gene therapy in rheumatoid arthritis (RA) and discuss recent progresses in the development of efficient silencing, focusing on expression of short hairpin RNAs. RESULTS/ CONCLUSIONS: Combining advances in RNAi methodology with gene therapy technology opens avenues for rapid applications to RA.
BACKGROUND: RNA interference (RNAi) is a powerful endogenous process initiated by short double-stranded RNAs, which results in sequence-specific posttranscriptional gene silencing. The possibility of blocking the expression of any protein carries huge expectations for potential therapeutic applications in a wide range of diseases. For clinical development, however, the use of RNAi-based therapeutics has to overcome major obstacles, mainly targeted delivery and safety issues. OBJECTIVE/ METHODS: In this short review, we provide an overview of specifications for RNAi-based gene therapy in rheumatoid arthritis (RA) and discuss recent progresses in the development of efficient silencing, focusing on expression of short hairpin RNAs. RESULTS/ CONCLUSIONS: Combining advances in RNAi methodology with gene therapy technology opens avenues for rapid applications to RA.
Authors: Evelina Miele; Gian Paolo Spinelli; Ermanno Miele; Enzo Di Fabrizio; Elisabetta Ferretti; Silverio Tomao; Alberto Gulino Journal: Int J Nanomedicine Date: 2012-07-20