Therapeutic RNAi Development: Targeted siRNA, qPCR Assays
RNA Interference (RNAi) technology is a relatively new but powerful tool to regulate gene expression and aid in the performance of loss-of-function research studies. The process of RNAi can occur naturally in the cell, and may have originally evolved from a primitive defense against viruses. RNAi consists of highly-selective small interfering RNA (siRNA) molecules utilized intracellularly by the RNA-induced silencing complex (RISC) to cleave the target mRNA and induce post-transcriptional gene silencing. As RISC recognizes and destroys double-stranded RNA as well as RNA that shares the double stranded RNA’s sequence, it is very effective at silencing specific genetic sequences, without affecting non-target genes. RNA interference precursors include small interfering RNA (siRNA), small hairpin RNA (shRNA), and microRNA (miRNA). When introduced by cell transfection in vitro and in vivo, siRNA results in effective silencing of mRNA expression in mammalian cells and tissues.
RNAi development has greatly improved the efficiency, specificity, and speed at which gene functions can be analyzed, playing a critical role in the drug discovery process. In mammalian cells, RNAi can be used to reduce the expression of specific genes in a sequence-specific approach, and it is a powerful method of identifying drug targets, as well as illuminating cell and biochemical signaling pathways.
This technology has great therapeutic potential for novel drug discovery. A number of pharmaceutical and biotechnology companies are investigating the possible use of siRNA-based molecules for inducing RNAi in vivo (particularly for use in animal models), and in RNAi-based therapeutics. Small RNA molecules (including siRNA, shRNA, and miRNA) are capable of targeting multiple misregulated cellular processes, therefore RNA interference therapeutics can be used to treat any disease associated with misregulation (typically overexpression) of specific genes. Numerous literature reports address potential application of RNAi to target genes involved in such diseases as cancer, Alzheimer’s, and a number of inflammatory and virally-associated diseases.
However, there are a number of technical difficulties associated with inefficient siRNA delivery into the cell and short siRNA stability time in vivo, therefore reduced biostability of unmodified RNA must be overcome. Stable and functional RNA molecules usually require chemical modifications and carrier molecule encapsulation (liposome-, lipid-, polymer-, etc) in combination with extensive experimental testing to ensure consistent functional effects, stability, and lack of off-target events. Therefore, Altogen Labs is focused on the development of efficient in vivo reagents and RNAi delivery technologies.
Our expertise in RNA interference allows us to provide complete A-to-Z gene silencing services and associated analytical assays, including qRT-PCR mRNA expression, Western Blot protein expression, functional cell-based assays, long-term RNA stability testing in vitro and in vivo, multiple RNA and DNA modifications (LNA, PNA), chemical modifications (2’Ome), incorporation of dyes, and much more.
At Altogen Labs, we recognize that providing fast delivery of products and services is critical to expediting research. We are committed to meeting our clients’ timely needs while maintaining high quality and integrity. All of our services undergo rigorous QC procedures.
Once we know the details of your project, we can provide you an immediate price quote (contact us via e-mail at firstname.lastname@example.org or call Altogen Labs at 512-433-6177). Please note that experimental details will help us to provide an accurate quote and timeline estimate.