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 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.
Researchers from the Polytechnic University of Catalonia have developed a technique that improves and cuts the cost of a technique called electroporation, which involves opening pores in cell membranes using an electric field to introduce substances like drugs and DNA. Current methods are aggressive and expensive whereas the new system manages to apply low voltage electroporation with a small printed circuit board, which costs less than a Euro per unit and does not damage cells.
Investigators with an interest in pre-clinical and translational musculoskeletal research are invited to attend the 3rd IOF-ESCEO Pre-Clinical Symposium to be held on April 18, in Rome, Italy.
New research suggests that lowering excessive levels of a protein in immune system cells could be a strategy to clear an infection that is deadly to patients with cystic fibrosis (CF).
For some time, researchers have known about disparities in diagnoses and outcomes among breast cancer patients based on race and age. However, they have been challenged to develop a set of criteria that can be used to reliably target drug delivery mechanisms based on an individual patient's tumor.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office has issued Notices of Allowance for three patent applications from the company's exclusively held Tuschl II patent estate.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of liver disease associated with AAT deficiency. These data were presented at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, "The Liver Meeting") held November 9-13, 2012 in Boston.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2012, and company highlights.
Alnylam Pharmaceuticals, Inc. and Genzyme, a Sanofi company, announced today that they have formed an exclusive alliance to develop and commercialize RNAi therapeutics targeting transthyretin (TTR) for the treatment of transthyretin-mediated amyloidosis (ATTR) in Japan and other Asia-Pacific countries.
The online journal PLOS ONE released a scientific report on the validation of Falcon Genomics' Cancer BioChip System (CBCS) as a tool for identifying inhibitors of breast cancer.
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