MicroRNA News and Research

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In genetics, microRNAs (miRNA) are single-stranded RNA molecules of 21-23 nucleotides in length, which regulate gene expression. miRNAs are encoded by genes from whose DNA they are transcribed but miRNAs are not translated into protein (i.e. they are non-coding RNAs); instead each primary transcript (a pri-miRNA) is processed into a short stem-loop structure called a pre-miRNA and finally into a functional miRNA. Mature miRNA molecules are partially complementary to one or more messenger RNA (mRNA) molecules, and their main function is to down-regulate gene expression.

Alnylam Pharmaceuticals to present results on RNAi therapeutics at ACS National Meeting

Alnylam Pharmaceuticals, Inc.,a leading RNAi therapeutics company, announced today that it will make a total of 16 oral presentations at the American Chemical Society (ACS) Fall 2010 240th National Meeting & Exposition being held in Boston, Mass. from August 22-26, 2010.

24 Aug 2010

Alpha-synuclein gene silencing effective for treating neurodegenerative disorders: Research

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, and collaborators at The Parkinson's Institute and the Mayo Clinic have published new research findings in the journal Public Library of Science (PLoS). The new data show effective silencing of the alpha-synuclein gene with an RNAi therapeutic administered directly to the substantia nigra in the CNS of non-human primates.

24 Aug 2010

MMV joins Pool for Open Innovation against neglected tropical diseases

The Medicines for Malaria Venture (MMV) today became the first product development partnership (PDP) to contribute intellectual property to the Pool for Open Innovation against Neglected Tropical Diseases. MMV joins GlaxoSmithKline (GSK), Alnylam Pharmaceuticals, and the Massachusetts Institute of Technology (MIT) in contributing to the Pool, which is administered by BIO Ventures for Global Health (BVGH).

17 Aug 2010

Study finds abnormal expression of miRNAs can contribute to human diseases

Researchers at Tufts University School of Medicine and Tufts Medical Center have identified an RNA sequence that promotes increased numbers of specific microRNAs (miRNAs), molecules that regulate cell growth, development, and stress response. The discovery helps researchers understand the links between miRNA expression and disease, including heart disease and cancer. The findings are published in the August 13 issue of Molecular Cell.

17 Aug 2010

MeCP2 regulatory protein determines vulnerability to cocaine addiction: Research

A regulatory protein best known for its role in a rare genetic brain disorder also may play a critical role in cocaine addiction, according to a recent study in rats, funded by the National Institute on Drug Abuse (NIDA), a component of the National Institutes of Health. The study was published today in the journal Nature Neuroscience.

16 Aug 2010

Rxi Pharmaceuticals second-quarter net loss decreases to $2.1 million

RXi Pharmaceuticals Corporation, a recognized leader in RNAi-based therapeutic discovery and development, today reported its financial results for the second quarter ended June 30, 2010.

11 Aug 2010

USPTO allows Regulus Therapeutics' claims of key Tuschl III patent covering Mir-155

Regulus Therapeutics Inc. announced today that the United States Patent and Trademark Office has allowed claims in a patent application (US Serial No. 12/550,579) derived from the "Tuschl III" patent series. The allowed claims relate to human microRNA miR-155, a microRNA that is a potential target for immuno-inflammatory indications, and a lead program of the Regulus-GSK Strategic Alliance.

11 Aug 2010

Silence Therapeutics receives patent for RNA silencing method

Silence Therapeutics plc announces the issuance of United States patent 7,772,203, titled "Methods and Compositions for Controlling Efficacy of RNA Silencing," by the United States Patent and Trademark Office (USPTO). The issued patent generally claims methods of enhancing RNA silencing with a double stranded RNA interference (RNAi) agent. The patent's RNA silencing method claims include coverage for the administering of a pharmaceutical composition containing short interfering RNA (siRNA), micro RNA (miRNA), pre-miRNA or short hairpin (shRNA) molecules. Silence believes the proprietary structural modification techniques covered in this patent will play a key role in increasing the potential therapeutic efficacy of RNAi therapeutics.

11 Aug 2010

Alnylam Pharmaceuticals reports significant progress in discovery of novel technologies for systemic delivery of RNAi therapeutics

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, and collaborators from the Massachusetts Institute of Technology (MIT), The University of British Columbia (UBC), AlCana Technologies, Inc., and Tekmira Pharmaceuticals Corporation, presented results from multiple ongoing research efforts regarding the discovery of novel technologies for the systemic delivery of RNAi therapeutics.

10 Aug 2010

Isis second-quarter pro forma net operating loss increases to $15.5 million

Isis Pharmaceuticals, Inc. today announced its financial results for the quarter ended June 30, 2010. The Company finished the second quarter of 2010 with a pro forma net operating loss (NOL) of $15.5 million and $17.1 million for the three and six months ended June 30, 2010, respectively, compared to a pro forma net operating loss of $1.3 million and pro forma net operating income of $798,000 for the same periods of 2009.

9 Aug 2010

Alnylam publishes research findings of Sort1 gene role in cardiovascular disease development

Alnylam Pharmaceuticals, Inc. a leading RNAi therapeutics company, announced today the publication of new research findings in the journal Nature describing the discovery and validation of the role of the gene Sort1 in the development of cardiovascular disease, including myocardial infarction (MI). This work was done with collaborators at University of Pennsylvania (UPenn) School of Medicine, Massachusetts General Hospital (MGH), and the Broad Institute. The collaborative effort combined genome-wide association studies (GWAS) and RNAi technology to identify and validate novel genes as targets for new therapies for heart disease.

6 Aug 2010

WaferGen launches SmartChip System for discovery, validation of biomarkers

WaferGen Biosystems, Inc., a leading developer of state-of-the-art genomic analysis systems, today announced it has launched the breakthrough WaferGen SmartChip Real-Time PCR System, the next-generation Real-Time PCR system for discovery and validation of biomarkers, or gene expression patterns, on a single platform. The SmartChip System is designed to provide accurate, highly sensitive and high-throughput gene expression profiling capabilities to researchers, clinicians and pharmaceutical companies.

6 Aug 2010

Genzyme, Isis announce results of two mipomersen phase 3 studies

Genzyme Corp. and Isis Pharmaceuticals Inc. today announced results of two phase 3 studies of mipomersen in patients who had high cholesterol levels while on maximally tolerated lipid-lowering therapy. In the study of patients with severe hypercholesterolemia, mipomersen reduced LDL-C, the primary endpoint, by 36 percent compared with a 13 percent increase for placebo.

5 Aug 2010

Alnylam second-quarter revenues increase to $26.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter ended June 30, 2010, and company highlights.

5 Aug 2010

Scientists identify tiny RNA molecule that increases blood stem cells

Investigators have identified a new mechanism that controls the number of hematopoietic stem cells - cells that give rise to all blood and immune system cells. In a report in the online Early Edition of Proceedings of the National Academy of Sciences, researchers from Massachusetts General Hospital (MGH) and the Harvard Stem Cell Institute identify a tiny RNA molecule that increases the number of these blood stem cells, an advance that may improve treatment of blood system cancers.

5 Aug 2010

Certain human leukemias can be treated by targeting stem-cell microRNAs: Research

Hematopoietic stem cells provide the body with a constant supply of blood cells, including the red blood cells that deliver oxygen and the white blood cells that make up the immune system. Hematopoietic-or blood-stem cells must also make more copies of themselves to ensure that they are present in adequate numbers to provide blood throughout a person's lifetime, which means they need to strike a delicate balance between self-renewal and development into mature blood-cell lineages. Perturb that balance, and the result can be diseases such as leukemia and anemia.

5 Aug 2010

Premier international conference to discuss latest diagnostic tools, emerging therapies for personalized medicine

What As scientists understand more about a tumor's underlying biology, personalized medicine is emerging as the new patient management paradigm. This year, the American Association for Cancer Research will host its fourth Molecular Diagnostics in Cancer Therapeutic Development: Challenges and Horizons conference at the Sheraton Denver Downtown Hotel in Denver, Colo. This meeting regularly draws representatives from academia and industry to discuss the latest in diagnostic tools and emerging therapies.

4 Aug 2010

miRagen Therapeutics develop MicroRNA based therapeutics for cardiovascular and muscle disease

miRagen Therapeutics, Inc., a biopharmaceutical company focused on improving patients' lives by developing innovative microRNA-based therapeutics for cardiovascular and muscle disease, announced today the publication of data demonstrating the essential role of microRNA-451 (miR-451) in the regulation of erythropoiesis, the process of red blood cell production in the body.

3 Aug 2010

Researchers discover manipulation of microRNA-29a shows potential of novel IBS treatment

A possible cause of irritable bowel syndrome has been traced to a small piece of RNA that blocks a substance protecting the colon membrane, leading to hostile conditions that can produce diarrhea, bloating and chronic abdominal pain.New research shows that this RNA segment sends signals that stop the activity of the gene that produces glutamine, an amino acid. Previous research has linked a shortage of glutamine in the gut with the seepage of toxins and bacteria through the intestinal wall, irritating nerves and creating disease symptoms.

3 Aug 2010

Scientists identify way to regulate uncontrolled growth of blood vessels

Researchers at the University of California, San Diego School of Medicine have identified a new way to regulate the uncontrolled growth of blood vessels, a major problem in a broad range of diseases and conditions.

2 Aug 2010

MicroRNA News and Research

Further Reading

Alnylam Pharmaceuticals to present results on RNAi therapeutics at ACS National Meeting

Alpha-synuclein gene silencing effective for treating neurodegenerative disorders: Research

MMV joins Pool for Open Innovation against neglected tropical diseases

Study finds abnormal expression of miRNAs can contribute to human diseases

MeCP2 regulatory protein determines vulnerability to cocaine addiction: Research

Rxi Pharmaceuticals second-quarter net loss decreases to $2.1 million

USPTO allows Regulus Therapeutics' claims of key Tuschl III patent covering Mir-155

Silence Therapeutics receives patent for RNA silencing method

Alnylam Pharmaceuticals reports significant progress in discovery of novel technologies for systemic delivery of RNAi therapeutics

Isis second-quarter pro forma net operating loss increases to $15.5 million

Alnylam publishes research findings of Sort1 gene role in cardiovascular disease development

WaferGen launches SmartChip System for discovery, validation of biomarkers

Genzyme, Isis announce results of two mipomersen phase 3 studies

Alnylam second-quarter revenues increase to $26.6 million

Scientists identify tiny RNA molecule that increases blood stem cells

Certain human leukemias can be treated by targeting stem-cell microRNAs: Research

Premier international conference to discuss latest diagnostic tools, emerging therapies for personalized medicine

miRagen Therapeutics develop MicroRNA based therapeutics for cardiovascular and muscle disease

Researchers discover manipulation of microRNA-29a shows potential of novel IBS treatment

Scientists identify way to regulate uncontrolled growth of blood vessels

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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