About siRNA
RNA Interference
RNAi, or RNA interference, is a newly discovered mechanism for RNA-guided regulation of gene expression in which double-stranded ribonucleic acid inhibits the expression of genes with complementary nucleotide sequences. This biological pathway can be used to silence the activity of specific genes, on a selective basis. For example, although proteins perform many vital functions in the human body, the over-abundance, hyperactivity or misplacement of a particular protein in the body can be harmful. RNAi can help target such proteins by selectively “silencing” the relevant gene, and thereby can be helpful in treating the disease. This scientific discovery has been considered so potentially groundbreaking that in 2006, two scientists, Dr. Andrew Fire of Stanford University School of Medicine and Dr. Craig Mello of the University of Massachusetts Medical School, were awarded the Noble Prize in Physiology or Medicine for their work in RNA interference.
The Science of RNA Interference and siRNA
SiRNA, or short interfering ribonucleic acid, comprises a class of 20-25 nucleotides that are double-stranded RNA molecules, and that interfere with (or “silence”) the expression of a specific gene. In addition to their role in the RNAi pathway, siRNA also can act as an antiviral mechanism or in the formation of the chromatin structure of a genome. Structurally, each siRNA strand is composed of a 5’ phosphate group and a 3’ hydroxyl group. Dicer, a ribonuclease in the RNase III family that contains two RNase III domains and one PAZ domain, acts as the cleaver of the double-stranded RNA (dsRNA), which is converted into siRNAs. These siRNA are the molecules that trigger RNA interference.
Moreover, with knowledge of the specific gene sequence, siRNA can be artificially placed in cells to target specific genes. Then, scientists can test the synthetic siRNAs to ascertain whether the siRNA silences the intended gene activity and stifles the protein’s synthesis.
The Future of siRNA™
Growing Demand for RNAi Therapeutics. We believe that the biotechnology and pharmaceutical industries may pursue increased RNAi-based therapeutic transactions in the future. The ability to suppress or silence specific, targeted genes in animal models may lead to the initial development of more potent drugs for humans. Based on our patent portfolio and siRNA expertise, we believe that the pharmaceutical and biotechnology industries may view us as a market leader and desirable partner.
Worldwide Market Potential. ZaBeCor’s market consists of potentially millions of patients with chronic diseases and a continuing need for medication both in the U.S. and the world. Based on the human genome base sequencing model, we believe that it may be possible to synthesize siRNA to silence potentially any protein in humans that can cause disease. If this proves correct, we believe the potential market demand for ZaBeCor’s products will be worldwide.
Substantial Therapeutic Advantages. Based on experimental data, we believe that a single molecule of siRNA effectively can perform the work of many molecules used in traditional drugs, which suggests that only one molecule of siRNA may be needed to silence the growth of many protein molecules. Based on our current research, we believe that the drugs that could be developed from our patented technologies may have minimal side effects and may have the potential to become the drug of choice, with substantial therapeutic advantages over current drugs in several diseases, including asthma and allergy. We have completed initial development testing, which, based on our preliminary results, has been effective in pre-clinical models.