Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
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.
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.
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.
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.
Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy.
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).
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.
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.
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.
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.
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.
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.
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.
Pancreatic ductal adenocarcinoma, the most common of pancreatic cancers, is extraordinarily lethal, with a 5-year survival rate of just 6 percent. Chemotherapy treatments are poorly effective, in part due to a high degree of drug-resistance to currently used regimens.
In developing antisense therapeutics, scientists at Ionis evaluate thousands of oligonucleotides. One of my responsibilities is to oversee the QC and management of oligonucleotides used in target screening.
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.
Bacteria that cause tuberculosis trick immune cells meant to destroy them into hiding and feeding them instead. This is the result of a study led by researchers from NYU Langone Medical Center and published online April 18 in Nature Immunology.
In an article published online ahead of print on Feb. 19, 2015 in the Journal of Biological Chemistry, investigators at the Medical University of South Carolina and the Ralph H. Johnson VA Medical Center report findings from in vitro and in vivo studies that elucidate the mechanisms underlying the impaired ciliogenesis and abnormal kidney development characteristic of polycystic kidney disease (PKD).
microRNAs (miRs) are small endogenous noncoding RNA molecules (20–23 nucleotides) derived from imperfectly paired hairpin RNA structures naturally encoded in the genome that act specifically as triggering molecules to control translational repression or mRNA degradation.
A drug that would be the first to target the cause of Huntington's disease (HD) is effective and safe when tested in mice and monkeys, according to data released today that will be presented at the American Academy of Neurology's 68th Annual Meeting in Vancouver, Canada, April 15 to 21, 2016.