Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA molecules, 20-25 nucleotides in length, that play a variety of roles in biology. Most notably, siRNA is involved in the RNA interference (RNAi) pathway, where it interferes with the expression of a specific gene. In addition to their role in the RNAi pathway, siRNAs also act in RNAi-related pathways, e.g., as an antiviral mechanism or in shaping the chromatin structure of a genome; the complexity of these pathways is only now being elucidated.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.
Silencing genes that have malfunctioned is an important approach for treating diseases such as cancer and heart disease. One effective approach is to deliver drugs made from small molecules of ribonucleic acid, or RNA, which are used to inhibit gene expression. The drugs, in essence, mimic a natural process called RNA interference.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.
Nanoparticles that deliver short strands of RNA offer a way to treat cancer and other diseases by shutting off malfunctioning genes. Although this approach has shown some promise, scientists are still not sure exactly what happens to the nanoparticles once they get inside their target cells.
A major study from researchers at the La Jolla Institute for Allergy and Immunology provides new revelations about the intricate pathways involved in turning on T cells, the body's most important disease-fighting cells, and was published today in the prestigious scientific journal Nature.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5.
These particles are all classed as inorganic particles, which can all be utilised in biomedical applications. They differ in terms of their inherent material and size dependent physiochemical properties, for example, their optical and magnetic properties.
Arcturus Therapeutics, Inc., an industry leader in RNAi technologies for the treatment of disease, today announced it has raised $1.3 million in a seed funding round led by multiple high net-worth private investors from the United States and Canada.
Researchers have developed a new drug delivery system that allows inhalation of chemotherapeutic drugs to help treat lung cancer, and in laboratory and animal tests it appears to reduce the systemic damage done to other organs while significantly improving the treatment of lung tumors.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.
Kinase inhibitors are molecules that block the activity of kinases. Kinases are a specific class of enzymes. They are extremely important in signal transduction processes in the human body meaning that they actually regulate most of the physiological processes that take place in the body.
RNA, once considered a bit player in the grand scheme by which genes encode protein, is increasingly seen to have a major role in human genetics.
Santaris Pharma A/S, a privately held biopharmaceutical company focused on developing medicines targeted to disease-related mRNAs and microRNAs, today announced a worldwide strategic alliance with Bristol-Myers Squibb to discover and develop novel medicines using Santaris Pharma's proprietary Locked Nucleic Acid Drug Platform.
A University of Alberta researcher is thinking small to find innovative ways to improve the delivery of drugs that can be more easily administered with fewer side-effects.
New classes of drugs that can silence specific genes, such as small interfering RNAs (siRNAs), offer great therapeutic potential. But the specific delivery of siRNAs to target cells to exert their effects remains a significant challenge. A novel nanoparticle-based approach that enables more efficient delivery of siRNA drugs is presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.
CanCELL cell lines are featuring gene knock-outs of the major cancer relevant oncogenes and tumor suppressor genes. This new product line answers identified market needs in oncology research and drug discovery.
DNA, siRNA and miRNA can reprogram cancer cells - that is, if these nucleic acids could cross through the cell membrane. A University of Colorado Cancer Center study published today in the journal Therapeutic Delivery shows that cholesterol "rafts" can shepherd genetic payloads into cancer cells.
Alnylam Pharmaceuticals a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).
By loading fragile RNA into silicon nanoparticles, researchers from The Methodist Hospital and two other institutions found a new drug delivery system can reduce the size of ovarian tumors by as much as 83 percent -- and stop tumor growth in chemotherapy-resistant ovarian cancer tissue.