Oligonucleotide News and Research

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A new combinatorial therapy for treatment of liver cancer

In the latest issue of Molecular Therapy, Skoltech and MIT researchers have published a new combinatorial therapy for the treatment of liver cancer.

20 Apr 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

Horizon Discovery and Pharmahungary report positive early-stage results for novel micro-RNA therapeutic for ischemic heart diseases

Horizon Discovery Group plc and Pharmahungary Group announced positive early-stage results for a novel micro-RNA therapeutic for ischemic heart diseases.

31 Mar 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

New partnership aims to explore, cultivate innovations in cell and gene therapy

Some patients who have not responded to traditional medicines are now experiencing remarkable recoveries thanks to next-generation immunotherapies. These therapies equip a patient's own immune cells to recognize, target, and destroy cancer cells.

25 Nov 2019

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

Adult SMA patients treated with Nusinersen show improvement in motor function

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.

11 Nov 2019

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

Pluripotent stem cells help advance understanding of how Huntington's disease develops

Huntington's disease (HD) is a fatal hereditary disease for which there is no cure. A novel study from Cedars-Sinai Medical Center, Los Angeles, using pluripotent stem cells advances understanding of how the disease develops and may help pave the way for identifying pathways for future treatments.

21 Oct 2019

Custom-made oligonucleotide therapy for girl with fatal Batten disease

Sufferers of fatal Batten disease have been given new hope as scientists develop a custom-made oligonucleotide therapy for a young girl.

14 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

Spatial Gene Expression Profiling of Neurological Disorders

In this interview, Cedric Uytingco discusses the key benefits of being able to visualize gene expression patterns in neurological samples.

From 10x Genomics, Inc. 7 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

Promising new drug being developed for polycystic kidney disease

Researchers have developed a potential new drug for the treatment of Polycystic kidney disease (PKD) – a deadly genetic condition affecting the kidneys that leads to formation of swellings or cysts within the kidney that cause it to fail.

12 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

IDT launches high quality, ready-to-use custom oligo pools

Continuing its mission to advance biological research worldwide, Integrated DNA Technologies has launched oPools Oligo Pools – ready-to-use pools of high quality DNA oligonucleotides (oligos) up to 350 nucleotides in length.

From Integrated DNA Technologies 16 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

HDO-antimiR could be a potent weapon to combat microRNA-related disease

MicroRNA (miRNA) is a type of RNA that plays an important role in various cellular processes and is also involved in developing defenses against pathogens. Increasing numbers of studies have indicated that certain human diseases are caused by altered expression and organization of miRNA.

27 Jul 2019