Antisense News and Research

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Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.

Researchers uncover THOR gene involved in cancer development

It turns out Thor, the Norse god of thunder and the Marvel superhero, has special powers when it comes to cancer too.

15 Dec 2017

New compound targets APOE gene to reduce Alzheimer's damage in mice

People who carry the APOE4 genetic variant face a substantial risk for developing Alzheimer's disease.

6 Dec 2017

Biogen and Ionis awarded 2017 Prix Galien USA Award for SPINRAZA

Biogen and Ionis have been awarded the prestigious 2017 Prix Galien USA Award for Best Biotechnology Product for SPINRAZA (nusinersen). The Prix Galien USA Award recognizes extraordinary achievement in scientific innovation that improves the state of human health. The award was presented at a ceremony in New York City on October 26, 2017.

30 Oct 2017

Trial shows safety and tolerability of antisense oligomer for treatment of influenza A

A phase 1 clinical trial published in the British Journal of Clinical Pharmacology found that radavirsen -- an antisense oligomer that inhibits the production of certain influenza proteins -- is safe and well-tolerated in healthy individuals.

20 Sep 2017

Exploration of human genetics reveals new targets to combat heart disease

Again and again, it's the rare among humans that help the rest of us. The exploration of human genetics is revealing new targets to combat heart disease among atypical variants. Mutations in genes that play a role in heart health are the inspiration for a cluster of new heart drugs.

7 Sep 2017

New drug shows promising results for treating spinal muscular atrophy

A drug developed by an Iowa State University biomedical researcher as a potential treatment for spinal muscular atrophy showed promising results in a recently published study.

26 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

Researchers solve medical mystery through genetics

Researchers have identified the genetic mutation responsible for one patient's serious health problems, finally solving a medical mystery that has endured for over 30 years.

6 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

Emory researchers reveal 'matchmaker' role for protein linked to SMA

A puzzling question has lurked behind SMA, the leading genetic cause of death in infants. The disorder leads to reduced levels of the SMN protein, which is thought to be involved in processing RNA, something that occurs in every cell in the body.

14 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

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy.

9 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

MDA celebrates FDA approval of new spinal muscular atrophy drug

The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants.

23 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