UCLA researchers have developed a novel delivery platform for efficient and specific gene silencing in RNAi therapy.
BACKGROUND:
RNA interference (RNAi) and microRNA are potent methods for efficient and specific gene silencing. The technologies have been extensively exploited to develop therapies for treating fatal disorders like cancer and neurogenetic disease, etc. However, micro-RNAs and small interfering RNAs (siRNAs) are highly susceptible to degradation by enzymes if delivered directly to cells by transfection, thereby limiting the number of copies that can be delivered. Alternatively, interfering RNAs can be delivered via retroviral or DNA viral vectors, but the genetic material is synthesized by a more complicated and less efficient process that involves trafficking in and out of the nucleus. Thus, to fully harness the power of RNAi technologies, novel approaches that allow stable, safe and effective delivery and synthesis of interfering RNAs are needed.
INNOVATION:
UCLA researchers have developed a novel delivery platform for efficient and specific gene silencing in RNAi therapy. The delivery vehicle involves an RNA polymerase and interfering RNA genome that allows efficient amplification of the RNA up to one-million-fold under the control of a subgenomic promoter. Additionally, unlike the RNA delivered by retrovirus that needs to be transcribed inside the nucleus and then processed by DICER into “pre” and “mature” micro-RNA, the synthesized RNA in this new technology is amplified in the cytoplasm and can be picked up directly by RISC for RNA interference.
POTENTIAL APPLICATIONS:
• RNAi therapy
ADVANTAGES:
• Allows the delivery of abundant copies of RNAi genomes
• Efficient generation of RNAi genomes without involving the nucleus
DEVELOPMENT-TO-DATE:
This invention has been developed and tested in vitro.