Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
Cancer cases have been rising over the years and according to the statistics, the number of people living with cancer will continue to increase.
Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences.
Researchers from the University of California San Diego School of Medicine have found a way to create new neurons in a mouse model of Parkinson's disease. The severely debilitating and progressive neurodegenerative disease, as of now, has no cure, and this breakthrough could help hundreds of thousands of patients with the condition feel experts.
Xiang-Dong Fu, PhD, has never been more excited about something in his entire career. He has long studied the basic biology of RNA, a genetic cousin of DNA, and the proteins that bind it. But a single discovery has launched Fu into a completely new field: neuroscience.
Now, in new research published on the preprint server bioRxiv European scientists have performed RNA structure probing to obtain a single-base resolution secondary structure map of the full SARS-CoV-2 coronavirus genome.
Using a first-of-its-class drug in a clinical trial, an international research effort headed by a scientist at University of California San Diego School of Medicine reports that inhibition of a key enzyme safely and effectively improved the health of persons with non-alcoholic fatty liver disease, a chronic metabolic disorder that affects hundreds of millions of people worldwide.
Antisense Therapeutics, an Australian biopharmaceutical company developing and commercializing antisense pharmaceuticals for unmet need in rare diseases announces a virtual poster presentation on the Muscular Dystrophy Association Virtual 2020 Conference website.
An international team led by Emory scientists has gained insight into the pathological mechanisms behind two devastating neurodegenerative diseases.
New research led by hearing scientists at Oregon Health & Science University suggests an avenue to treat and prevent intractable genetic disorders before birth.
Seizure disorders in babies are frightening and heartbreaking. A new basic science breakthrough offers hope for a potential treatment for rare developmental and epileptic encephalopathies resulting from a single genetic mutation.
Over half of our genomes are made of repeating elements within DNA. In rare cases, these repeats can become unstable and grow in size. These repeat "expansions" cause neurodegenerative diseases such as ALS and dementia as well as learning disorders and autism in Fragile X syndrome.
A genetic mutation that disrupts how DNA sends messages to the rest of a cell has been linked to a large number of blood cancers.
Aging is a complex and natural process that affects all living organisms. As people age, normal biological processes are affecting, resulting in a decline in various organs and age-related conditions. Though aging is normal, some children affected by a disease called Progeria may age way faster than others.
A study published in the Journal of Neuromuscular Diseases presents the first evidence of mild improvement or stabilization of motor and respiratory function in adults with spinal muscular atrophy type 3 (SMA3) treated with Nusinersen, which was the case even in patients who have had the disease for 20 years or more.
Jennifer J. Lentz, PhD, Associate Professor at LSU Health New Orleans Neuroscience Center of Excellence and Departments of Otorhinolaryngology, Genetics and Ophthalmology, in collaboration with Robert K. Koenekoop MD, PhD and Professor of Pediatric Surgery, Human Genetics and Ophthalmology at McGill University in Montreal, Quebec, Canada, has been awarded a $1.74 million USD grant to advance research on Usher syndrome.
An unprecedented case at Boston Children's Hospital shows that it's possible to do something that's never been done before: identify a patient's unique mutation, design a customized drug to bypass it, manufacture and test the drug, and obtain permission from the Food and Drug Administration (FDA) to begin treating the patient -- all in less than one year.
A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy.
Researchers from the Josep Carreras Leukemia Research Institute, discover that a non-coding region of the genome originates a key molecule for the proliferation of tumors in breast cancer and some types of sarcoma.
Hepatoblastoma is a rare form of liver cancer affecting just a few individuals per million. However, it is the leading cause of liver cancer in infants and young children, with most patients diagnosed before their third birthday.
People with familial chylomicronemia syndrome are born with a genetic mutation that means they can't produce an enzyme called lipoprotein lipase.