RNAi is a new technology which aims to target the aspect of mRNA, which is a type of genetic product. It is a natural system which works in plants and animals to knock out the production of a certain gene product.
Here’s how RNAi works.
The cells in your body create proteins and other substances through the transcription and translation of DNA. The DNA, which stays inside the safe center called the nucleus, sends out messages saying what to make in the form of RNA.
RNAi works by targeting a specific string of RNA. So if you have RNAi targeting the production of “protein-18alpha,” when the RNAi sees the message for “protein-18alpha” inside the cell, it goes to it and destroys it.
This specific targeting allows for the knockout of specific proteins.
There is great potential in this area for a variety of therapeutic treatments. As you can imagine, being able to specifically stop the production of a certain product inside the cell offers many advantages.
It could, for instance, in theory be used to stop the production of elements needed for the replication of viruses such as HIV.
HIV needs to make a lot of envelope proteins, for instance, to coat itself as its particles leave the cell. If you could knock out that envelope protein’s production, you could then prevent the HIV from spreading. RNAi in theory could do so.
RNAi has potential in the treatment of many conditions including cancer and high cholesterol. If there is a genetic product that is associated with sickness or disability, then in theory and increasingly in study, there is the potential of RNAi as a treatment.
But while RNAi has great potential in the treatment of many conditions, there have yet to be any major breakthroughs due to its use. The reason for this lies in the nature of the substance.
RNAi is a large, complex substance that is damaged and degraded if taken orally by itself. Additionally, it is not able to travel well inside of cells.
It is small enough to be removed from the body by renal clearance, yet too large and charged to travel inside cells well.
Delivery issues are one of the most important factors preventing RNAi from achieving significant therapeutic effect. Yet research continues in that area to improve it.
Ideas to improve delivery of RNAi have covered a wide range. Coupling with antibodies to aid delivery is one approach. Another is use of nanoparticles, particularly coupling with aptamers, or highly charged nucleic acids selected for affinity to their target.