Gene Silencing

Gene silencing is an epigenetic alteration of gene expression that causes formerly active individual genes or larger chromosomal regions to become inactive. Changes in DNA methylation levels and changes in covalent modifications of histone proteins cause chromatin compaction, making genes inaccessible to the transcription machinery. Posttranscriptional gene silencing can occur due to mRNA degradation and/or translation suppression. Small RNA regulators such as small interfering RNAs (siRNAs), microRNAs (miRNAs), and Piwi-associated RNAs (piRNAs), which are created from diverse kinds of double-stranded RNA (dsRNA) accumulating in cells, are generally responsible for these effects. Small RNAs, particularly siRNAs, can also play a role in gene silencing at the transcriptional level.

Gene Silencing

Gene silencing is a relatively recent therapy method that uses the body’s natural mechanisms to regulate disease by inhibiting or silencing certain genes linked to specific disorders.

Gene silencing may be accomplished in a variety of ways. Silence Therapeutics is focused on RNA interference, a biological phenomenon. All of our bodies are born with an RNA interference function meant to regulate gene activity or defend the body against viruses.

Gene silencing isn’t the same as gene therapy. Gene Therapy treatment is infusing new DNA into the body to supplant an absent or breaking down quality. It is a reversible, controlled impact, which has benefits if there are any negative effects. It’s also quite particular and focused, which ensures that the outcomes are predictable.

It is a rapidly developing field of study. It has already been authorized for several disorders and has the potential to give highly-tailored therapies for specific hereditary diseases as a potential lifetime therapy. 

A study of the anatomy of a cell helps understand gene silencing. In the human body, what is the structure of a cell?

In the human body, there are around 30-40 trillion cells. Each cell has an exterior membrane (layer), cytoplasm (a jelly-like material), and a nucleus (a central core that regulates the rest of the cell’s activities). The nucleus houses DNA (deoxyribonucleic acid), which holds the ‘genetic code’ of living organisms.

Gene silencing therapy

The cell production process might become incorrect at times, resulting in errors. For example, parts of DNA might be missing from the genetic instructions, or cells could start to mutate (change). Cells may create too much of a certain protein or produce a protein that isn’t working properly. This can result in many hereditary illnesses, such as cystic fibrosis, Huntington’s disease, thalassemia, and cancer. Some of these illnesses are difficult to treat using traditional methods. For genetically based diseases, gene silencing therapy might be a viable option.

RNAi

In the late 1990s, RNA interference (RNAi) was a game-changing discovery in molecular biology. In 2006, the scientists who discovered it was awarded the Nobel Prize. It’s a natural regulation mechanism that’s part of the immune response. The immunological response is the body’s defence mechanism against foreign invaders such as viruses, bacteria, and parasites.

RNAi molecules remove mutations, fix errors, and combat viruses that have penetrated DNA. They have a role in the synthesis of new proteins, but they may also function in the opposite direction, modifying or altering the activity of genes as necessary. By ‘neutralizing’ and lowering the function of particular mRNA molecules, they limit or prevent gene expression. This is an example of RNAi in action.

Little interfering RNAs are formed when strands of RNA are split or ‘diced’ into small pieces during RNAi (siRNA). This tight spot to mRNA targets keeps them from acting, beginning the RNAi cycle. This’silences’ or ‘turns down the synthesis of the infection-related protein permitting the cell to get back to its generally expected sound condition.

Role of RNAi in Gene Silencing Technology

The goal of gene silencing is to intervene in the process of gene expression before it reaches the stage of translation. The RNAi process is triggered when an ‘anti-code’ intended to disrupt troublesome mRNA is introduced, allowing certain disease-associated genes to be silenced (temporarily switched off).

Gene silencing molecules are made to look like siRNA, allowing them to connect to particular sections of a gene in the same manner that siRNA does, preventing the gene’s message from being transmitted. The main problem is efficiently and safely distributing the siRNA-mimicking molecules to the target cells.

Gene silencing agents work by temporarily suppressing a gene’s messenger, halting or reversing illness progression by focusing on the underlying disease process rather than the symptoms it creates.

Conclusion

We now understand how gene silence renders genes inert. However, even in plants, it might have negative consequences. As a result, gene modification, gene editing, and gene silencing procedures require prior approval.

Scientists are nearing completion on a new gene silencing method for Huntington’s disease mediated by RNA interference. In addition, many methods for various genetic illnesses are now in the research phase.