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.
Avidity NanoMedicines LLC, a biopharmaceutical company pioneering a breakthrough approach to the targeted delivery of nucleic acid-based medicines, announced today that its scientific co-founder, Mark E. Davis, Ph.D., will receive the 2014 Prince of Asturias Award for Technical and Scientific Research.
The suppression of two genes reduce breast cancer tumor formation and metastasis by interfering with blood vessel formation and recruitment, report scientists from Houston Methodist and five other institutions in the Proceedings of the National Academy of Sciences (now online).
A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder.
RNA interference (RNAi) is a naturally occurring process, which cells can use to silence, or ‘turn off’ unwanted genes.
Working together, Johns Hopkins biomedical engineers and neurosurgeons report that they have created tiny, biodegradable “nanoparticles” able to carry DNA to brain cancer cells in mice.
Scientists at The University of Texas at Austin have discovered that a protein produced by the influenza A virus helps it outwit one of our body's natural defense mechanisms. That makes the protein a potentially good target for antiviral drugs directed against the influenza A virus.
Scientists at Indiana University have unlocked one of the mysteries of modern genetics: how acquired traits can be passed between generations in a process called epigenetic inheritance. The new work finds that cells don't know to silence some genes based on information hardwired into their DNA sequences, but recognize heritable chemical marks that are added to the genes. These chemical tags serve as a form of molecular memory, allowing cells to recognize the genes and remember to silence them again in each new generation.
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it received regulatory approval to begin a Phase 2a clinical trial of ARC-520, its RNAi-based drug candidate for the treatment of chronic hepatitis B virus (HBV) infection.
Inspired by tiny particles that carry cholesterol through the body, MIT chemical engineers have designed nanoparticles that can deliver snippets of genetic material that turn off disease-causing genes.
Forget the oysters and the champagne this Valentine's Day. If you want to keep your true love's heart beating strong, the real foods of love are dark chocolate and red wine, said Loyola University Health System preventive heart specialist Sara Sirna, MD.
Researchers from Spain have identified several new splicing events regulated by the oncogenic splicing factor SRSF1 in lung cancer, suggesting that their dysregulation is involved in the pathogenesis of the disease.
Gene related to the proliferation of cancerous cells blocked through molecular technology. A group of researchers from Mexico's General Hospital, Health Secretariat, Medicine Faculty and the Institute of Cellular Physiology of the National Autonomous University of Mexico (UNAM) identified a therapeutic target for cervix cancer: gene CDKN3.
Resistance of tumor cells toward multiple cytostatic drugs is a serious problem in cancer treatment. In the journal Angewandte Chemie, a team of Chinese and American researchers has now introduced a new approach to gene therapy that could counter this problem: The gene that codes for resistance is “silenced” through the use of an ingenious nanocomplex.
A team of researchers led by an NIBIB grantee at Vanderbilt University has created a biodegradable scaffold that enables sustained, local delivery of gene-silencing factors called siRNA to promote tissue regeneration. The team recently used the scaffold to successfully deliver siRNA to mice in order to locally silence a gene normally responsible for inhibiting blood vessel formation.
Horizon Discovery and AstraZeneca have entered into a research, collaboration and license agreement to explore a range of oncology-relevant genotypes with the aim of identifying and validating a number of novel drug targets. This deal is the second collaboration between AstraZeneca and Horizon, and follows the announcement in April 2013 of an oncology discovery program to explore Horizon's first-in-class kinase target program, HD-001.
A novel breast-cancer therapy that partially reverses the cancerous state in cultured breast tumor cells and prevents cancer development in mice, could one day provide a new way to treat early stages of the disease without resorting to surgery, chemotherapy or radiation, a multi-institutional team led by researchers from the Wyss Institute of Biologically Inspired Engineering at Harvard University reported January 1 in Science Translational Medicine.
Ikaria, Inc., a critical care company focused on developing and commercializing innovative therapies designed to address the significant needs of critically ill patients, announced today that its investor group and employee shareholders have reached a definitive agreement to sell the Commercial Business to Madison Dearborn Partners in a transaction valued at approximately $1.6 billion.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated a pilot Phase II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene in development for the treatment of TTR-mediated amyloidosis.
For the first time, large-scale information on the biochemical makeup of small interfering RNA (siRNA) molecules is available publicly. These molecules are used in research to help scientists better understand how genes function in disease.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans.
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