Enhancing Gene Silencing by RNAi

NU 2005-063, NU 2005-064, and NU 2006-182

 

Inventors

Richard Carthew*
Dinari Harris
Young Sik Lee

 

Short Description

Novel strategy to improve gene silencing by RNAi for development of therapeutics

 

Abstract

RNAi has been proposed as a therapeutic strategy for disorders characterized by aberrant gene overexpression (cancer, macular degeneration etc.) as well as for controlling viral infections. However, the main roadblock for using RNAi technology as a therapeutic is that several human proteins normally inhibit this process, lowering the efficiency of gene silencing. Northwestern researchers have devised a method for identification of these proteins and have identified several endogenous RNAi inhibitory proteins. Prof. Carthew and colleagues are conducting experiments to strategically inhibit these proteins in order to enhance RNAi efficiency. By identifying and disabling RNAi inhibiting host proteins, RNAi could evolve into an effective treatment for various diseases. The search for RNAi inhibitory proteins has also been undertaken in animal cells, suggesting that this approach could enable improvements in production of genetically modified organisms for research purposes.

 

Applications

  • Gene therapy for infectious or chronic diseases
  • Rational drug design
  • RNAi laboratory research

 

Advantages

  • Improved RNAi efficiency
  • Applicable to a wide range of host cells

 

Publications

Lee YS, Pressman S, Andress AP, Kim K, White JL, Cassidy JJ, Li X, Lubell K, Lim do H, Cho IS, Nakahara K, Peall JB, Bellare P, Sontheimer EJ, Carthew RW (2009) Silencing by Small RNAs is Linked to Endosomal Trafficking, Nature Cell Biology. 11: 1150-1156.

 

IP Status

US patents 8,318,688 and 8,735,371 have been issued. International patent application has been filed.  

Patent Information: