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.

LSU Health New Orleans awarded $1.74 million to advance research on Usher syndrome

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.

22 Oct 2019

Researchers develop customized drug treatment to bypass patient’s unique mutation

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.

11 Oct 2019

Experimental treatment shows early promise to help nearly half of DMD patients

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.

25 Sep 2019

Researchers discover a molecule that regulates tumor development in different types of cancers

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.

5 Sep 2019

Breast cancer gene could be key to developing targeted therapy for hepatoblastoma

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.

4 Sep 2019

Researchers test new drug to treat patients with chylomicronemia syndrome

People with familial chylomicronemia syndrome are born with a genetic mutation that means they can't produce an enzyme called lipoprotein lipase.

9 Aug 2019

Prion disease in mice treated successfully with antisense oligonucleotides

Prion proteins are known to cause scrapie – a neurodegenerative condition. It is capable of debilitating damage to the nervous system. Researchers have successfully devised a treatment for this condition which prolonged the lives of the lab mice infected with the prions.

1 Aug 2019

New drug provides hope for patients with Duchenne muscular dystrophy

The results from three clinical trials have shown that a new drug can successfully delay the progression of Duchenne muscular dystrophy.

10 Jul 2019

Scientists aim to develop new ointment that could reduce risks of primary skin cancer

Scientists from Far Eastern Federal University, V.I. Vernadsky Crimean Federal University, Dmitry Mendeleev University of Chemical Technology, and Far Eastern Branch of the Russian Academy of Sciences, assumed the risks of primary skin cancer and its recurrences can be significantly reduced by applying the ointment with antisense oligonucleotides which are short DNA, RNA fragments used in oncology to suppress the synthesis of tumor proteins.

3 Jul 2019

Researchers discover underlying cause of hereditary muscle disease

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.

27 Mar 2019

RNA-induced gene fusion can occur in mammalian cells, reveals study

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.

29 Jan 2019

New class of antisense oligonucleotide therapeutics tested in humans

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.

28 Jan 2019

New therapy for childhood blindness shows 'very promising' results

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.

18 Dec 2018

New mouse model shows potential for rapid identification of promising muscular dystrophy therapies

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.

13 Dec 2018

First bifacial molecule can invade double-stranded DNA or RNA

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.

13 Nov 2018

New form of therapy may stop or reverse progressive vision loss

A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness.

1 Nov 2018

Newly developed synthetic DNA molecule may one day be used as 'vaccine' for prostate cancer

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.

19 Oct 2018

Researchers develop two-pronged approach for targeting Ebola virus

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.

12 Oct 2018

Krembil scientists develop potential treatment to stop knee and spine osteoarthritis

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.

5 Oct 2018

Blocking stress-responsive protein reduces loss of tumor suppressor gene in breast cancer

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.

4 Oct 2018