Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
Researchers at Karolinska Institutet have discovered the underlying cause of a hereditary muscle disease first characterised in a Swedish family in 1980. It proves to be the first identified disease caused by defective myoglobin, the protein that transports oxygen in muscle cells.
A fusion gene is a new gene made by joining parts of two different genes. The current thought is that fusion genes can happen in cells with unstable genome when part of the DNA from one chromosome moves to another chromosome.
A first-in-human study with a new class of antisense oligonucleotide therapeutics showed the ability to target the RNA-silencing drug to the liver, resulting in improved potency and safety at therapeutic doses. The design and results of this trial, conducted in healthy human volunteers are reported in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.
A new therapy aimed at improving the sight of people with one of the most common forms of childhood blindness, has shown 'very promising' initial results, according to a study involving UCL researchers.
A Massachusetts General Hospital research team has created a new mouse model of a common form of muscular dystrophy with the potential of rapidly distinguishing promising therapeutic drugs from those unlikely to be successful.
Carnegie Mellon University researchers have developed a synthetic molecule that can recognize and bind to double-stranded DNA or RNA under normal physiological conditions. The molecule could provide a new platform for developing methods for the diagnosis and treatment of genetic conditions.
A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness.
Researchers from City of Hope, a world-renowned comprehensive cancer center and independent biomedical research institution, have developed a synthetic DNA molecule that is programmed to jump-start the immune system to eradicate genetically distinct types of prostate cancer.
In a new study researchers have developed a two-pronged approach for targeting Ebola virus infection using linked nucleic acid (LNA) antisense oligonucleotides (ASOs)designed to interfere both genes essential for translation of Ebola virus genes and to block production of an intracellular human protein needed for the virus to enter cells.
Scientists at the Krembil Research Institute have developed a novel therapeutic treatment that has the potential to stop knee and spine osteoarthritis in its tracks.
Over half of all breast cancers carry genetic defects in the p53 gene, a powerful tumor suppressor. Loss of this gene increases the risk of getting breast cancer and the resultant cancers become highly resistant to treatment.
New research from the University of British Columbia suggests that reducing mutated Huntington disease protein in the brain can restore cognitive and psychiatric impairments in mice.
Pediatric researchers have identified a gene mutation that causes a serious lymphatic condition, and used that knowledge to restore normal lymphatic vessels in model animals. The laboratory findings may lead to a new therapy for patients with this type of abnormal lymphatic circulation.
The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.
NIH-funded researchers delayed signs of amyotrophic lateral sclerosis in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1.
PureTech Health plc, a clinical-stage biopharmaceutical company developing novel medicines focused on the Brain-Immune-Gut Axis, today announced that it has entered into a multiyear collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., to advance PureTech's milk-derived exosome platform technology for the oral administration of Roche's antisense oligonucleotide platform.
Spinal muscular atrophy is a genetic disease that affects motor neurons in the spinal cord, resulting in muscle atrophy and widespread weakness that eventually impair swallowing and breathing.
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.