RNAi Therapeutics

RNAi Therapeutics

RNAi (RNA interference) is a process or mechanism that is found in all living cells and is responsible for inhibiting the “expression” or further development or behaviors of specific genes. This inhibition, in turn, affects the production, or lack thereof, of certain proteins. Also known as therapeutic gene silencing, RNAi research has come far in the past decade. Since its discovery that was published in Nature journal in 2001, greater understanding regarding the processes and potential of RNAi technologies in the treatment of diseases has continued. RNAi technologies are now focusing on state-of-the-art and innovative treatments and therapies in the treatment of a number of disease processes including cancers, infectious diseases, and obesity. In the medical and pharmaceutical industry, the focus on the development of effective drugs that specifically target a particular gene may provide huge strides in the treatment, and even potential reversal, of certain disease processes. In addition, the ability to specifically target genes that cause cellular damage or destruction leading to disease processes enables pharmaceuticals and therapeutics to specifically target genes that have previously been unaffected by a number of traditional drug therapies.

At its most basic definition, RNAi technologies potentially utilized for the treatment of diseases can quite effectively “silence” or halt the expression or development and production of genes that cause disease (such as oncogenes). The degree of specificity in targeting such genes is another advantage of RNAi therapeutic modalities, as is the ability to utilize numerous RNA sequences in a specific or singular drug product in targeting and delivering drugs or medicines to cell-specific tissues or organisms. The ultimate goal for researchers is to find ways to damage, slow down, or kill defective and cancerous cellular proliferation without damaging or otherwise affecting surrounding tissues. Numerous patents in the process of further developing siRNA drugs and pharmaceuticals or small molecules and antibodies have been filed by biotechnology and pharmaceutical companies globally. Targeting RNAi-based therapeutics in the treatment of infectious diseases such as hepatitis, metabolic conditions such as obesity, and cancerous tumor growth is the focus of many companies who are sponsoring novel RNAi therapeutics through pre-clinical research efforts and clinical trials.

Research scientists use RNAi biological phenomena to study particular genes and their functions. The main hurdle in using RNAi for therapeutic gene silencing is the effective intracellular delivery of siRNA into the cell or tissue. The capability of siRNAs to mediate post-transcriptional gene silencing in mammalian cells and tissues, as well as its successful use to prevent expression of target mRNA has recently led to the development of a new methodology for novel drug discovery.

Several pharmaceutical and biotechnology companies are currently investigating the possible use of synthetic siRNA for inducing RNAi in vivo, its use in animal models, and in RNAi-based therapeutics. Small RNA molecules, such as siRNA, shRNA, andmicroRNA have been regarded as potential therapeutic agents to target multiple misregulated cellular processes therefore it is theoretically possible that RNAi can be utilized to treat any disease associated with over expression of specific genes. In fact, there are many reports in the literature that address the potential therapeutic application of RNAi to specifically target genes involved in multiple diseases including various forms of cancer, Alzheimer’s, and a number of inflammatory and virally-associated diseases.

However, a number of major difficulties associated with inefficient delivery of functional RNA moleculesinto cells and the reduced biostability of unmodified RNA must be overcome. Therefore, our research is focused on the development of efficient in vivo reagents and RNAi delivery technologies. Efficient and organ-specific delivery of synthetic oligonucleotide molecules is currently a key limiting step to enable siRNA- and microRNA-based therapeutic approaches.

In Vivo Transfection Kits from Altogen Biosystems

RNAi has been used for in vivo target validation studies using animal models. The major challenge in performing RNAi studies in vivo is the effective, directed delivery of functional siRNA molecules into specific tissues. Altogen® in vivo transfection reagents could be conjugated with siRNA and administered systemically via intravenous (i.v) tail vein injection in order to provide directed gene silencing in specific tissues, including liver, pancreas, kidney, and tumors. Selective knockdown could be seen as early as 24 hours after injection.

Featured articles:

siRNA Transfection || RNAi || siRNA Library Screening || In Vivo Transfection Reagents || Pre-optimized Transfection Kits || Cell Transfection

Featured transfection products from Altogen Biosystems:

LNCaP Cell Transfection Kit || In Vivo Nanoparticle Transfection Kit || In Vivo PEG-Liposome Transfection Kit

Altogen Biosystems is a life sciences company focused on development, marketing and manufacture of cell type specific transfection products. Efficient delivery of DNA, RNA, and siRNA enabled by advanced formulation of reagents and peculiar design of protocols. Altogen Biosystems offers a complete transfection system for over 80 cell lines, as well as hard-to-transfect and primary cells. All reagents are functionally tested to be highly reproducible, serum compatible, induce low toxicity, and can be used for co-transfection experiments, and high throughput applications.

RNAi Services by Altogen Labs CRO:

Altogen Labs provides all types of RNAi laboratoty services, as well as other specialized biotechnology and pharmaceutical services, including generation of stable cell lines, RNA interference (RNAi) services, pharmacology and toxicology testing: IC-50, assay development, siRNA library screening and transfection services, liposome encapsulation, cell banking and cryopreservation services.