Oligonucleotides News and Research

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Oligonucleotides are short sequences of nucleotides (RNA or DNA), typically with twenty or fewer bases. Automated synthesizers allow the synthesis of oligonucleotides up to 160 to 200 bases.

DNA sensor systems provide highly sensitive monitoring of cancer-related enzyme activity

The development of DNA sensor systems is of great importance for advances in medical science. Now another piece of the puzzle for the development of personalized medicine has been found with the results of a highly sensitive monitoring of cancer-related topoisomerase II enzymes.

24 Aug 2017

Positive CHMP opinion recognizes efficacy of Biogen’s SPINRAZA for treatment of spinal muscular atrophy

Biogen today announced the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending the granting of a marketing authorization for SPINRAZA to treat patients with spinal muscular atrophy (SMA).

21 Apr 2017

Targeted treatment slows progression of two degenerative brain diseases in mice

Scientists report a significant step toward combatting two degenerative brain diseases that chip away at an individual's ability to move, and think.

12 Apr 2017

Using Spherical Nucleic Acids to Track and Treat Disease

Spherical nucleic acids are structures that are made by taking a nanoparticle template and using chemistry to arrange short strands of DNA or RNA on the surface of those particles. The spherical core of the nanoparticle creates a spherical arrangement of DNA or RNA, similar to tiny little balls of nucleic acids.

From Pittcon 1 Mar 2017

MIT researchers develop new machine to rapidly produce customized peptides

Manufacturing small proteins known as peptides is usually very time-consuming, which has slowed development of new peptide drugs for diseases such as cancer, diabetes, and bacterial infections.

27 Feb 2017

Designer compound may be effective tool for tackling tau-related disorders

In a study of mice and monkeys, National Institutes of Health funded researchers showed that they could prevent and reverse some of the brain injury caused by the toxic form of a protein called tau.

8 Feb 2017

Research unravels molecular mechanisms of antisense oligonucleotides

Stanley T. Crooke, MD, PhD, CEO of Ionis Pharmaceuticals and recipient of the 2016 Lifetime Achievement Award from the Oligonucleotide Therapeutic Society presents a detailed look at the mechanisms that underlie antisense drug activity in the article entitled "Molecular Mechanisms of Antisense Oligonucleotides," published in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

2 Feb 2017

Synthetic molecule appears to reverse Alzheimer's-related neurological damage

Under ordinary circumstances, the protein tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that damage brain cells.

25 Jan 2017

CSHL researcher-inventor hails FDA approval of new SMA drug

Within a week of Christmas day, a drug called nusinersen will be in the hands of doctors across the nation, who will use it, most urgently, to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA).

26 Dec 2016

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has approved SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

26 Dec 2016

Turning off 'fat-specific protein 27' improves blood sugar levels, reduces body fat in animal model

In a study published in the Journal of Lipid Research, Saint Louis University scientist Angel Baldan, Ph.D., reports that turning off a protein found in liver and adipose tissue significantly improves blood sugar levels, as well as reduces body fat in an animal model.

16 Dec 2016

Study offers promise for therapeutic management of fetal and congenital genetic diseases in utero

A breakthrough study by research teams at Rosalind Franklin University of Medicine and Science and Oregon Health & Science University offers promise for therapeutic management of congenital diseases in utero using designer nucleotide sequences that can simply be injected into the fluid surrounding the developing fetus to potentially treat disabling-to-lethal genetic defects.

7 Oct 2016

Scientists find way to boost efficiency with which CRISPR-Cas9 cuts and disables genes

CRISPR-Cas9 is the go-to technique for knocking out genes in human cell lines to discover what the genes do, but the efficiency with which it disables genes can vary immensely.

18 Aug 2016

Overcoming gene silencing barriers to target neurological conditions: an interview with Dr Errol de Souza

Many gene mutations that cause neurological disorders have been identified. For example, in a rare neurological disorder such as Huntington’s disease, an autosomal dominant mutation through expansion of CAG (cytosine-adenine-guanine) triplet repeats in the gene coding for the Huntingtin protein results in abnormal protein production.

1 Aug 2016

Novel conjugate therapy shows promise in destroying ALL cells with few side effects

Researchers at UC Davis and Ionis Pharmaceuticals have developed a hybrid treatment that harnesses a monoclonal antibody to deliver antisense DNA to acute lymphoblastic leukemia (ALL) cells and that may lead to less toxic treatments for the disease.

29 Jul 2016

Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.

25 Jul 2016

Duke to lead early-stage clinical trials for promising new neurological therapies

Duke University could receive up to $19 million to lead early-stage clinical trials for new drugs to treat neurological conditions such as Alzheimer's disease and neuropathy.

22 Jul 2016

Researchers discover novel neuroprotection strategies to slow progression of Parkinson's disease

Using a robust model for Parkinson's disease, University of Alabama at Birmingham researchers and colleagues have discovered an interaction in neurons that contributes to Parkinson's disease, and they have shown that drugs now under development may block the process.

20 Jul 2016

Automated Liquid Handling in Pharmaceutical Research

In this interview, News Medical talks to Dr. Alfred Chappell of Ionis Pharmaceuticals about automated liquid handling in pharmaceutical research.

From Beckman Coulter Life Sciences - Automation and Genomics 26 May 2016

Researchers develop novel ss-siRNAs to turn off disease-related genes

Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity.

13 May 2016