RNA interference (RNAi) is a natural process within cells that regulates the activity of genes. It involves the use of small RNA molecules to inhibit the expression of specific genes. When RNAi is triggered, these small RNA molecules, known as small interfering RNA (siRNA) or shRNA, bind to messenger RNA (mRNA), preventing it from being translated into protein or targeting it for degradation. This mechanism plays a crucial role in various biological processes, including the regulation of gene expression, defense against viruses, and development. Scientists have also harnessed RNAi for research purposes and potential therapeutic applications, such as treating genetic disorders and infectious diseases.
siRNA is a type of small RNA molecule that plays a key role in the regulation of gene expression. It is involved in a natural cellular process known as RNA interference (RNAi), which serves to silence or downregulate specific genes.
siRNA molecules are typically 20-25 nucleotides long and are designed to be complementary to a target gene's messenger RNA (mRNA). When introduced into a cell, siRNA molecules specifically bind to the target mRNA, triggering its degradation or inhibiting its translation into a protein. This process can effectively "silence" the expression of the targeted gene, allowing researchers to study gene function or potentially develop therapeutic applications.
RNAi technology has broad applications in research, including the study of gene function, the identification of potential drug targets, and the development of gene therapies to treat various diseases, including genetic disorders and certain types of cancer. Currently, our science team is developing siRNA drugs for COVID and seasonal influenza.
The key differences between mRNA (messenger RNA) and
siRNA (small interfering RNA) lies in their roles and functions within cells
In the world of genetics, small RNA molecules like siRNA and microRNA play a fascinating role in controlling gene activity. They can essentially turn genes on or off at different stages of gene expression, from the initial reading of the DNA code to the final protein production. This knowledge has profound implications for our understanding of gene regulation and provides a potential method for manipulating gene functions in the future. Small RNA molecules like siRNA have the incredible power to fine-tune gene expression in human cells. By targeting specific genes and regions, scientists can potentially harness this power for various applications in developing effective treatments for diseases.
The research team at Intelligene is dedicated to exploring how siRNA impacts gene expression. We are developing innovative siRNA drugs for precise treatments of infectious diseases. Our commitment is to unlock tomorrow's cures and enhance human health.
IG-001 and IG-002 are our first-in-class antiviral drugs targeted to SARS-CoV-2 and flu.
These RNA therapeutics target and degrade the viral RNAs inside the infected cell, essentially killing the virus.
The siRNA-based therapeutics can be delivered a multitude of ways.
either inhaled,intravenous or via the skin with micro-needle patches
When it comes to gene therapy, we must discuss how nucleic acid drugs can be effectively delivered and function in the human body. Unlike traditional medications, nucleic acid drugs, such as siRNA, encounter numerous barriers, both extracellularly and intracellularly, from the moment they enter the body until they reach their target cells. Unprotected RNA is inherently unstable under physiological conditions and is susceptible to enzymatic degradation by endogenous nucleases, often leading to its breakdown before reaching the target cells, significantly compromising therapeutic efficacy.
One significant application of lipid nanoparticles (LNPs) and exosome-based cell encapsulation technologies is overcoming the challenge of enabling RNA drugs to traverse these formidable barriers within the human body, ultimately reaching their target cells for effective treatment. Intelligene's siRNA drugs will harness LNP and exosome encapsulation technologies, aiming to maximize therapeutic benefits for various diseases.
