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.

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

UCSF scientists build largest molecular interaction map for autism

For more than two decades, researchers have identified hundreds of genes that increase the risk of autism spectrum disorder (ASD).

28 Aug 2026

New study identifies key pathway for delivering antisense cancer drugs

Antisense oligonucleotides (ASOs) have been proposed as a therapeutic approach to shut down the production of faulty proteins in a wide variety of diseases, including cancer.

11 Aug 2026

RNA-based therapeutic strategy targets brain pathway behind cancer-associated cachexia

A new path has opened toward stopping cancer-associated cachexia, a devastating complication that gradually wastes patients away.

2 Aug 2026

Rice University chemists develop cheaper way to synthesize drugs

Pharmaceutical drugs often rely on chemical compounds found in the body. Take phosphate, a common compound cells use as a chemical switch.

29 Jul 2026

Tailored gene therapy dramatically reduces childhood epilepsy seizures in trial

SCN2A‑related developmental epileptic encephalopathy (DEE) is a rare, severe form of childhood epilepsy and one of the most common causes of monogenic autism.

21 Jul 2026

Blocking ZFP384 protein extends brain repair window after stroke

Blocking the activity of ZFP384 can prolong the brain’s endogenous repair functions after stroke, report researchers in a collaborative study.

13 Jul 2026

Antisense oligonucleotides reverse rare neurodevelopmental disorder in preclinical models

Scientists at St. Jude Children's Research Hospital have found they can reverse the effects of HNRNPH2-related neurodevelopmental disorder using antisense oligonucleotides (ASOs) in preclinical models. ASOs are short synthetic nucleic acid strands that target specific messenger RNA.

22 Apr 2026

Early genomic testing prevents years of inconclusive visits for pediatric patients

For many families, eight years of uncertainty meant repeated specialist visits, inconclusive genetic tests and the growing fear that a clear answer might never come. For nearly half of the children enrolled in the Telethon Undiagnosed Disease Program (TUDP), that wait is now over.

21 Apr 2026

NTU Singapore and partners develop new inhaled treatment for severe lung infections

Nanyang Technological University, Singapore (NTU Singapore) is working with China's Southern University of Science and Technology (SUSTech) and Swedish biotechnology company Lipigon Pharmaceuticals AB to develop a new inhaled treatment aimed at helping patients recover faster from severe lung infections.

13 Apr 2026

Pseudogene lncRNAs regulate cancer stem cell behavior and signaling

Cancer stem cells (CSCs), a critical subpopulation within tumors, drive cancer initiation, progression, metastasis, relapse, and resistance to therapy due to their innate capacity for self-renewal and differentiation.

11 Apr 2026

Novel compound clears harmful protein, protects neurons in frontotemporal dementia models

New research from Washington University School of Medicine in St. Louis adds to growing evidence that helping brain cells break down and eliminate their own cellular waste is a promising treatment strategy for a variety of neurodegenerative diseases.

31 Mar 2026

New initiative aims to scale individualized therapies for rare diseases

Critical Path Institute (C-Path) today announced the launch of One to Millions, a global, multi-stakeholder public-private initiative to enable scalable development of advanced therapies for highly individualized conditions.

26 Mar 2026

Targeting protein fragment may improve Huntington disease treatments

Treatments that target a fragment of the mutant protein that causes Huntington's disease might be more effective than treatments, now in clinical trials, that target the whole protein but leave this fragment intact, a new study in mice suggests.

18 Mar 2026

Researchers find novel path to treat Rett syndrome

A team of researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new approach to treat Rett syndrome – offering early promise for a rare neurodevelopmental disorder that currently has no cure.

5 Mar 2026

Reducing miRNA levels helps rescue vascular defects in Rett syndrome

MIT researchers have discovered that two common genetic mutations that cause Rett syndrome each set off a molecular chain of events that compromises the structural integrity of developing brain blood vessels, making them leaky.

25 Feb 2026

Novel antisense oligonucleotide shows promise against aggressive pancreatic cancer

Pancreatic ductal adenocarcinoma (PDAC) is the most lethal form of pancreas cancer. It's also the most common form of the disease.

8 Jan 2026

Tau biology expands beyond microtubules to neurodegeneration, metabolism and mental illness

An Thought Leaders Invited Review published today in Genomic Psychiatry by Dr. Peng Lei and colleagues presents a sweeping synthesis of tau protein research that fundamentally reframes our understanding of this molecule's dual identity.

16 Dec 2025

Study reveals how TDP-43 causes neuronal overactivity in ALS and FTD

A new Northwestern University study using patient nervous tissue and lab-grown human neurons has uncovered how a key disease protein, TDP-43, drives overactive nerve cells in the neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

31 Oct 2025

Eating peanuts while pregnant may influence how a child’s genes respond to breastfeeding

Maternal peanut and peanut butter consumption during pregnancy and breastfeeding shaped DNA methylation patterns in children, particularly in genes regulating neurodevelopment and inflammation. These findings suggest maternal diet can prime offspring epigenetic sensitivity to breastfeeding duration.

27 Oct 2025