Oligonucleotide News and Research

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Researchers identify novel therapeutic approach for most frequent genetic cause of ALS

A team of scientists led by researchers from the University of California, San Diego School of Medicine and Ludwig Institute for Cancer Research have identified a novel therapeutic approach for the most frequent genetic cause of ALS, a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontal temporal degeneration, the second most frequent dementia.

10 Nov 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

New method to monitor activity of gene promoters during response to drug

When developing new drugs, monitoring cellular responses to candidate compounds is essential for assessing their efficacy and safety. Researchers from the RIKEN Center for Life Science Technologies report a new method to monitor and quantify the activity of gene promoters during the response to a drug, using the advanced gene expression analysis method CAGE followed by single-molecule sequencing.

3 Oct 2013

Chemists Find New Way To Put The Brakes On Cancer

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10 Sep 2013

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

A research team at Cold Spring Harbor Laboratory has used a recently developed technology they call TSUNAMI to create the first animal model of the adult-onset version of spinal muscular atrophy, a devastating motor-neuron illness.

10 Sep 2013

Novel machine learning-based approach used to predict hepatotoxic potential of DNA drug

New classes of therapeutic antisense oligonucleotides can have toxic effects on the liver. A novel machine learning-based approach used to predict the hepatotoxic potential of an antisense drug based on its chemical sequence is presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

28 Aug 2013

ASO may correct striatal transcriptional abnormalities and improve behavioral problems in HD mice

Findings from postmortem studies of the brains of Huntington's Disease (HD) patients suggest that transcriptional dysregulation may be an early step in the pathogenesis of HD before symptoms appear. Other studies report transcriptional alterations in the brains of some mouse models of HD.

27 Aug 2013

NanoString announces Early Access Program for nCounter Elements used in translational research

NanoString Technologies, Inc. (NASDAQ: NSTG), a provider of life science tools for translational research and molecular diagnostic products, today announced an Early Access Program for nCounter Elements™, a line of General Purpose Reagents (GPRs) developed specifically to meet the needs of translational research and clinical laboratories.

From NanoString Technologies 2 Aug 2013

Technique for developing more targeted drugs using molecular "robots"

Many drugs such as agents for cancer or autoimmune diseases have nasty side effects because while they kill disease-causing cells, they also affect healthy cells. Now a new study has demonstrated a technique for developing more targeted drugs, by using molecular "robots" to hone in on more specific populations of cells.

29 Jul 2013

Study suggests modulation of "enhancer-directed RNAs" can alter gene expression in living cells

In a pair of distinct but complementary papers, researchers at the University of California, San Diego School of Medicine and colleagues illuminate the functional importance of a relatively new class of RNA molecules.

5 Jun 2013

Novel technique for manufacturing short, single-stranded DNA molecules

A new method of manufacturing short, single-stranded DNA molecules can solve many of the problems associated with current production methods. The new method, which is described in the scientific periodical Nature Methods, can be of value to both DNA nanotechnology and the development of drugs consisting of DNA fragments.

3 Jun 2013

Safer, more effective treatment option for ovarian cancer

A novel, targeted approach to chemotherapy that makes ovarian cancer cells more susceptible to the cytotoxic effects of an antitumor drug may offer a safer, more effective treatment option for this often deadly form of cancer.

31 May 2013

Study results may help introduce better preoperative treatment for breast cancer

Breast cancers contain many different cell types with different patterns of gene expression, but a new study provides reassurance that this variability should not be a barrier to using gene expression tests to help tailor cancer treatments to individual patients.

3 May 2013

Santaris Pharma, Bristol-Myers Squibb partner to develop novel medicines using LNA Drug Platform

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.

18 Apr 2013

Roche, Isis Pharmaceuticals to develop neurodegenerative therapeutics for Huntington's disease

Roche and Isis Pharmaceuticals, Inc today announced that they have formed an alliance to develop treatments for Huntington's disease based on Isis' antisense oligonucleotide technology.

9 Apr 2013

Clinical trial signals new era in treatment of neurodegererative disorders

The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis - blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease - may be a first step towards a new era in the treatment of such disorders.

4 Apr 2013

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.

18 Mar 2013

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease.

25 Feb 2013

Non-coding RNA MALAT1 influences lung cancer metastasis

The vast majority - approximately 80 percent - of our DNA does not code for proteins, yet it gets transcribed into RNA. These RNA molecules are called non-coding and fulfill multiple tasks in the cell. Alongside a well-studied group of small RNAs, there is also a class of so-called long non-coding RNAs consisting of more than 200 nucleotides.

6 Feb 2013

Finding medical applications for unused inventions: an interview with Gabriel Mecklenburg, Co-Founder of Marblar

Marblar is gamifying the process of technology transfer. We post technology on our platform in the form of a challenge, and the Marblar community then competes to uncover new problems these technologies could solve – thereby discovering new market applications for these technologies.

31 Jan 2013