Antisense News and Research RSS Feed - Antisense News and Research

Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

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. [More]
CSHL researcher-inventor hails FDA approval of new SMA drug

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

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. [More]
MDA celebrates FDA approval of new spinal muscular atrophy drug

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

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. [More]
Study offers promise for therapeutic management of fetal and congenital genetic diseases in utero

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

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. [More]
Novel conjugate therapy shows promise in destroying ALL cells with few side effects

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. [More]
Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

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. [More]
Innovative mouse model could provide new strategy to arrest growth of pancreatic cancer

Innovative mouse model could provide new strategy to arrest growth of pancreatic cancer

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. [More]
Automated liquid handling in pharmaceutical research: an interview with Dr Alfred Chappell

Automated liquid handling in pharmaceutical research: an interview with Dr Alfred Chappell

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. [More]
Researchers develop novel ss-siRNAs to turn off disease-related genes

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. [More]
Study describes precise mechanisms that enable TB bacteria to persist in the body

Study describes precise mechanisms that enable TB bacteria to persist in the body

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. [More]
Researchers elucidate mechanisms underlying impaired ciliogenesis in PKD

Researchers elucidate mechanisms underlying impaired ciliogenesis in PKD

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). [More]
Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

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. [More]
New drug shows promise against Huntington's disease

New drug shows promise against Huntington's disease

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. [More]
Matched ChIP-validated Antibodies & Primer Sets

Matched ChIP-validated Antibodies & Primer Sets

Chromatrap announces that its high quality ChIP-validated antibodies can now be supplied with optimised matching primer sets. [More]
Scientists discover role of nucleocytoplasmic transport in specific forms of ALS and FTD

Scientists discover role of nucleocytoplasmic transport in specific forms of ALS and FTD

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are two devastating adult-onset neurodegenerative disorders. [More]
Novel drug shows promise against metastatic breast cancer in mouse models

Novel drug shows promise against metastatic breast cancer in mouse models

A doctor treating a patient with a potentially fatal metastatic breast tumor would be very pleased to find, after administering a round of treatment, that the primary tumor had undergone a change in character - from aggressive to static, and no longer shedding cells that can colonize distant organs of the body. [More]
U-M research could lead to new ways of fighting X-linked diseases in girls and women

U-M research could lead to new ways of fighting X-linked diseases in girls and women

Nearly every girl and woman on Earth carries two X chromosomes in nearly every one of her cells -- but one of them does (mostly) nothing. That's because it's been silenced, keeping most of its DNA locked up and unread like a book in a cage. [More]
Advertisement
Advertisement