Antisense News and Research

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Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.

Researchers develop new therapeutic approach to treat CLN3 Batten disease

Researchers led by a team at Rosalind Franklin University of Medicine and Science have devised a new therapeutic approach to treating a rare but deadly neurodegenerative genetic disease in children.

31 Jul 2020

Neurotransmitter-derived lipidoids help carry drugs across the blood-brain barrier in mice

Biomedical engineers at the Tufts University School of Engineering have developed tiny lipid-based nanoparticles that incorporate neurotranmitters to help carry drugs, large molecules, and even gene editing proteins across the blood-brain barrier and into the brain in mice.

24 Jul 2020

Study: L-Grb2 has therapeutic efficacy in preclinical models of ovarian and uterine cancer

The cover for issue 29 of Oncotarget features, "In vivo effects of treatment with L-Grb2 in combination with anti-angiogenic therapy in an ovarian tumor model," by Lara, et al. which reported that adaptor proteins such as growth factor receptor-bound protein-2 play important roles in cancer cell signaling.

23 Jul 2020

Arcturus and Duke-NUS announce approval of clinical trials for COVID-19 vaccine candidate

Arcturus Therapeutics Holdings Inc., a leading clinical-stage messenger RNA medicines company focused on the discovery, development and commercialization of therapeutics for rare diseases and vaccines, and Duke-NUS Medical School, Singapore's flagship research-intensive graduate entry medical school, today announced that the Clinical Trial Application for COVID-19 vaccine candidate LUNAR-COV19 has been approved to proceed by the Singapore Health Sciences Authority.

22 Jul 2020

Experimental drug for rare ALS shows promise in early clinical trial

An experimental drug for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a phase 1/phase 2 clinical trial conducted at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston and other sites around the world and sponsored by the pharmaceutical company Biogen Inc.

8 Jul 2020

Structure of the full SARS-CoV-2 RNA genome in infected cells

The current COVID-19 pandemic caused by a single-stranded RNA virus, thought to have jumped across species barriers to infect humans, has spread rapidly across the globe infecting over 10 million individuals. The virus, now known as the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), is the causative agent of COVID-19 disease. COVID-19 disease is a respiratory illness where symptoms can range from very mild to severe and include fever, coughing, a sore throat, and shortness of breath.

2 Jul 2020

Researchers develop a potential treatment approach for Pelizaeus-Merzbacher disease

A team led by Case Western Reserve University medical researchers has developed a potential treatment method for Pelizaeus-Merzbacher disease (PMD), a fatal neurological disorder that produces severe movement, motor and cognitive dysfunction in children.

1 Jul 2020

Scientists discover mechanism involving key protein that predicts cancer progression and relapse

Cancer cases have been rising over the years and according to the statistics, the number of people living with cancer will continue to increase.

30 Jun 2020

Researchers develop potential new therapy for common form of muscular dystrophy

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.

29 Jun 2020

Parkinson's disease breakthrough seen in mouse model

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.

25 Jun 2020

New therapeutic approach for Parkinson’s and other neurodegenerative diseases

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.

25 Jun 2020

Mapping RNA sequences of therapeutic value in the SARS-CoV-2 genome

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.

17 Jun 2020

Novel antisense drug shows promise to improve health of people with NAFLD

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.

16 Jun 2020

Antisense Therapeutics: Poster presentation on the Muscular Dystrophy Association Virtual Conference 2020 website

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.

15 Jun 2020

Scientists gain new insight into the pathological mechanisms behind two neurodegenerative diseases

An international team led by Emory scientists has gained insight into the pathological mechanisms behind two devastating neurodegenerative diseases.

6 May 2020

Research suggests an avenue to treat, prevent genetic disorders in the womb

New research led by hearing scientists at Oregon Health & Science University suggests an avenue to treat and prevent intractable genetic disorders before birth.

6 Apr 2020

New breakthrough offers hope for potential treatment of rare childhood epilepsy

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.

20 Feb 2020

Fragile X syndrome-causing repeats help human neurons function, study finds

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.

17 Feb 2020

Researchers reveal mutation's role in blood cancers

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.

3 Feb 2020

Potential new therapy for progeria

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.

18 Nov 2019