Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
In what researchers are calling a game changer for future ataxia treatments, a new study showed the ability to turn down the disease progression of the most common dominantly inherited ataxia.
A new approach to killing C. difficile that silences key bacterial genes while sparing other bacteria may provide a new way to treat the most common hospital-acquired bacterial infection in the United States, according to researchers.
Scientists at Cold Spring Harbor Laboratory have identified a therapeutic RNA molecule that corrects the error in genetic processing that leads to familial dysautonomia, a rare inherited neurodegenerative disorder.
According to new research, the toxin-antitoxin (TA) system found within E. coli can be induced to inhibit the growth of the bacterium. This may have important consequences for human infection.
A group of researchers at Osaka University reported the function of GTSF1 in male germ cells. The study, which can be read in EMBO Reports, shows that GTSF1 is an essential factor for secondary piRNA biogenesis by regulating piRNA-mediated cleavage of target RNA.
A proof-of-concept study carried out at UCL Institute of Ophthalmology with funding from Fight for Sight has highlighted the potential of a targeted antisense oligonucleotide therapy to treat Fuchs endothelial corneal dystrophy patients with a mutation in the TCF4 gene.
The first direct comparison of in vitro and in vivo screening techniques for identifying nanoparticles that may be used to transport therapeutic molecules into cells shows that testing in lab dishes isn't much help in predicting which nanoparticles will successfully enter the cells of living animals.
Because liver cancer is particularly diverse, genetically, and prone to relapse, identifying biomarkers that can predict disease progression is a critical goal in the fight against it.
By turning down the activity of a specific RNA molecule researchers at Sahlgrenska Academy, Sweden, have cured lung tumors in mice by 40-50 percent.
Mutations on a single gene, the huntingtin gene, are the cause of Huntington's disease. They lead to an incorrect form of the correspondent protein.
Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today in the New England Journal of Medicine.
A study from a team of Massachusetts General Hospital (MGH) investigators points toward a potential strategy for treating X-linked disorders - those caused by mutations in the X chromosome - in females.
It turns out Thor, the Norse god of thunder and the Marvel superhero, has special powers when it comes to cancer too.
People who carry the APOE4 genetic variant face a substantial risk for developing Alzheimer's disease.
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.
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.
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.
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.
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).
Scientists report a significant step toward combatting two degenerative brain diseases that chip away at an individual's ability to move, and think.