In recent years, RNA molecular biology has advanced phenomenally. In 2006, the Nobel Prize in Physiology or Medicine was awarded jointly to Andrew Z. Fire and Craig C. Mello for their discovery of RNA interference (RNAi) – gene silencing by double-stranded RNA.
According to the Nobel Prize:
‘They receive the prize for their discovery that double-stranded RNA triggers suppression of gene activity in a homology-dependent manner, a process named RNA interference (RNAi).
Their discovery revealed a new mechanism for gene regulation, and the biochemical machinery involved plays a key role in many essential cellular processes. Double-stranded RNA synthesized within the cell can reduce or abolish gene activity by RNAi-like mechanisms.
This control system for gene expression has proven to be important for both the development of an organism and the physiological functions of cells and tissues. Furthermore, RNAi protects against RNA virus infections, especially in plants and invertebrate animals, and secures genome stability by keeping mobile elements silent.
Today, double-stranded RNA is used as a powerful tool to experimentally elucidate the function of essentially any gene in a cell. The discovery of RNAi has already had an immense impact on biomedical research and will most likely lead to novel medical applications in the future’.
What is siRNA and how does it work
RNA (Ribonucleic Acid) is the messenger that carries instructions from the DNA to control the combination of proteins. RNA is found in all living cells. siRNA – small interfering RNA is sometimes known as short interfering RNA, or silencing RNA, and is essentially a form of double-stranded RNA molecules.
siRNA are approximately 21 nucleotides long, with 3 overhangs at each end which do the interfering of the translation of the proteins. siRNA works by binding to and encouraging the degradation of messenger RNA (mRNA) at specific sequences along the chain. By degrading the mRNA, siRNA prevents the production of certain proteins and therefore alters the combination of these proteins.
The importance of siRNA
The groundbreaking discovery that strands of RNA can selectively silence genes, has been one of the most important discoveries in biology, in recent years. This new finding has dramatically altered the way we view genetics, allowing new insights into cellular behaviour, and giving fresh motivation to those conducting medical research.
The potential for siRNA to be utilised in the treatment of previously incurable illnesses: viral infections, dominant disorders, cancer and neurological disorders shows great promise.
A recent study published by Celixir has shown a promising cancer gene therapy programme using siRNA. The in vitro study highlighted the potential of Celixir’s novel cancer gene therapies tp target solid tumours. Celixir is currently developing four siRNA molecules with the potential to be utilised as novel cancer gene therapies (programme CLXR-005).
The point is, Celixir wouldn’t be where they are today, on the brink of the next breakthrough in the fight against cancer, without the 2006 Nobel Prize winning technology – siRNA.