Articles tagged with Oligonucleotides
Researchers validated panels of antibodies targeting clinically relevant nucleic acid modifications to visualize antisense oligonucleotides in both in vitro and in vivo studies. The tools enable detection of modified nucleic acids irrespective of sequence, facilitating multiple clinical and pre-clinical workflows.
A UC San Francisco-led study found that delivering medicine for spinal muscular atrophy (SMA) via the amniotic fluid was safe and helped prevent damage to nerve cells. The therapy used molecules called antisense oligonucleotides (ASOs), which can alter gene expression, and was tested in mice and sheep with promising results.
Researchers at Tokyo University of Science have made breakthroughs in delivering gene-targeting compounds to the brain, using cholesterol-modified oligonucleotides that can penetrate the cerebral cortex beyond the blood vessels. This could lead to new treatments for diseases such as Alzheimer's and Parkinson's, as well as brain cancers.
The article discusses the need for bioanalytical assays to measure immune responses to oligonucleotide therapeutic drugs, especially when they include carriers or conjugates. Highly specific antibodies may enhance the development and production of ONTs, expanding studies on their safety and efficacy.
Researchers from Tokyo Medical and Dental University demonstrate a proof of concept for antisense nucleic acid therapy to prevent the spread of α-synuclein pathologies in synucleinopathies. The treatment, involving antisense oligonucleotides, effectively reduces Lewy pathology-like neuronal inclusion by over 90%.
A new study published in Nucleic Acid Therapeutics found that siRNA reduces huntingtin mRNA levels in the cytoplasm but not in the nucleus of mouse brains, suggesting a limitation in its effectiveness for treating Huntington's disease. The research highlights the importance of understanding the structure and function of nuclear RNA to ...
Researchers at Osaka University have developed molecules that can correct improper splicing of a vital tumor suppressor gene in neuroendocrine cancers. The study demonstrates that these splice-switching oligonucleotides can significantly reduce viable cancer cells and tumor size in mice, suggesting a novel therapeutic approach for intr...
Researchers developed a modified sugar that increases the effectiveness and safety of antisense oligonucleotides, a treatment strategy for central nervous system disease. The modification, called BNAP-AEO, decreases toxic side effects while improving gene silencing in brain cancer cells and mice.
Researchers from Osaka University have discovered a way to deliver antisense oligonucleotides to their targets inside cancer cells by opening specific calcium permeable channels. The new compound, L687, promotes efficient uptake of ASO into cancer cells, suppressing target gene activity and enhancing ASO efficacy.
Christiana Wang, a second-year PhD candidate, has been awarded the prestigious award for her platform presentation on antisense oligonucleotide therapy for a dominant negative SPTAN1 pathogenic variant. Her research aims to develop individualized therapy for treating rare genetic disorders.
A novel synthesis method enables easy linkage of therapeutic oligonucleotides to peptide markers, streamlining the process and making it more accessible and cost-effective. This breakthrough has the potential to produce more effective and targeted RNA-based drugs.
Researchers have developed a sustainable and scalable method to produce therapeutic oligonucleotides, which have the potential to treat various diseases. The new approach uses polymerases to amplify a catalytic DNA template in a single step, addressing challenges associated with current methods.
Researchers found that MALAT1 inhibition decreased BRAF RNA and protein levels, while increasing correlation with MAPK-associated genes. MALAT1-ASO treatment also reduced melanoma cell growth and tumor size in xenograft models.
Researchers developed predictive models to design RNA-binding inhibitors for disease treatment by regulating protein production. The study identified three key features for effective oligonucleotides: thermostability, serum resistance, and RNase H sensitivity.
Researchers uncover a pathophysiological mechanism that initiates and sustains neuropathic pain in mice, identifying Tiam1 as a potential therapeutic target. Targeting spinal Tiam1 with antisense oligonucleotides alleviates neuropathic pain hypersensitivity, offering promise for treating chronic pain.
Researchers at Cold Spring Harbor Laboratory have developed a potential therapeutic for diffuse intrinsic pontine glioma (DIPG) using antisense oligonucleotide technology. The treatment has slowed tumor growth, reversed changes in cancer cells, and increased survival rates in mice with DIPG.
A recent study found that the side effects of treating brain diseases with antisense oligonucleotides are related to altered calcium balance. By modulating calcium levels, researchers hope to reduce neurotoxicity and improve treatment outcomes for various neurological diseases.
The article reviews current terminal sterilization processes for oligonucleotide drug products and provides recommendations for formulation development and container closure selection. Terminal sterilization improves sterility assurance compared to membrane sterilization, but limitations exist.
Researchers from Japan have developed an RNA interference method using antisense oligonucleotides to correct a genetic defect in Fukuyama Muscular Dystrophy. This approach has shown promise in treating patients with the disease, which is characterized by generalized muscle weakness and intellectual disability.
A new treatment approach using antisense oligonucleotides (ASOs) may help reduce cystic fibrosis symptoms and improve quality of life for patients with a specific gene mutation. The ASO strategy tricks cells into making an imperfect but functional version of the CFTR protein, which is better than having none at all.
Researchers have discovered pairing Spinraza with valproic acid (VPA) can boost its therapeutic effects without increasing toxicity. This approach allows for improved SMN protein production in SMA patients, leading to longer survival and better muscle function.
Researchers at Aarhus University have developed an easy and inexpensive method for linking molecules to DNA sequences with desired functions. The method uses sulfonyl azides to introduce various functionalities, avoiding the need for expensive and unstable special phosphoramidites.
Non-viral mRNA and pDNA delivery systems have shown promising results in vaccine development against COVID-19 and are being explored for therapeutic applications. These systems aim to improve upon current formulations by enhancing stability, targeting specific tissues, and stimulating innate immune responses.
Researchers at Colorado State University are developing an RNA-based method for controlling herbicide-resistant weeds using gene-silencing technology. The goal is to create a non-genetically modified, shelf-stable spray that targets specific strands of RNA in weed cells, leaving crops untouched.
Scientists create a hybrid technology called heteroduplex oligonucleotide (HDO) that can safely and effectively silence disease-causing genes in certain immune cells. The HDO delivery method has shown promise in improving symptoms of autoimmune disorders and cancers by regulating the function of T and B lymphocytes.
Codiak BioSciences' exoASO-STAT6 demonstrates potent anti-tumor efficacy by reprogramming tumor-associated macrophages to an M1 phenotype, showing promise as a monotherapy candidate for hepatocellular carcinomas and other cancers. The company plans to initiate Phase 1 clinical trials in the first half of 2022.
Scientists at Cold Spring Harbor Laboratory have developed a way to interfere with the energy pathway that allows liver cancer to grow and spread by targeting the pyruvate kinase protein. This approach uses antisense oligonucleotides, which reduce tumor development in mouse models, offering a potential treatment for liver cancer.
Researchers at Rosalind Franklin University have identified a new therapeutic approach for treating cystic fibrosis. The treatment uses antisense oligonucleotides to restore CFTR function by removing stop mutations. This strategy has shown promise in treating CF patients with class I mutations and similar types of mutations.
Researchers developed an experimental drug that silences a faulty FUS gene, potentially treating rare and aggressive forms of ALS. The treatment delayed motor neuron degeneration in mice and showed promise in a patient with FUS-ALS.
Researchers at the Max Delbrück Center have developed a therapeutic agent to improve treatment of heart failure with preserved ejection fraction. The new approach targets alternative splicing in cardiac disease, using antisense oligonucleotides to stabilize sensitive molecules and trigger desired response.
A new study provides preliminary data for a human clinical trial of a treatment that targets the root cause of Alexander disease. The treatment has shown promising results in halting the progression of the disease and even reversing some symptoms in rat models, which better represent the human condition.
Researchers at IOCB Prague have created a glowing DNA enzyme called Supernova, which catalyzes a chemiluminescent reaction. This breakthrough uses artificial evolution to identify light-producing deoxyribozymes in a vast library of DNA molecules, opening up new possibilities for point-of-care assays and high-throughput screens.
Researchers reveal stable phosphatidylglycerol-DNA complex formation with strong van der Waals and hydrophobic interactions. The complex's structural parameters are determined, providing insight into the differences between DNA-phospholipid interaction and fatty acid binding.
Researchers developed new chemistry for antisense oligonucleotides to treat SMA, a debilitating genetic disease. The compounds demonstrate reduced toxicity and potential for longer-lasting treatment with fewer injections needed.
Researchers at UNC School of Medicine developed an improved oligonucleotide therapy strategy that can correct gene defects underlying cystic fibrosis. The approach has shown promising results in both human cells and mice, with potential applications for treating other pulmonary diseases.
Researchers at Penn Medicine have developed an RNA therapy that reversed blindness in a patient with a rare genetic disorder, improving vision over a 15-month period after a single injection. The treatment's durability and effectiveness provide new avenues for treating other ciliopathies.
Researchers have developed ultra-small nanomedicines that stably deliver oligonucleotides to refractory cancers, such as brain tumors and pancreatic cancer. These nanomedicines use Y-shaped block copolymers and nucleic acid drugs, achieving high permeability in cancer tissues and stability in the bloodstream.
Antisense oligonucleotides (ASOs) target disease-causing proteins, but can affect non-targeted proteins causing side effects. Researchers have developed a DNA/DNA double-stranded oligonucleotide that enhances ASO efficacy and stability in the body.
A first-in-human study found that MRG-110 significantly reduced miR-92a levels in healthy humans after a single intravenous dose. This inhibition has shown beneficial effects in animal models, including improved vascularization after myocardial infarction and accelerated wound healing.
Researchers at Karolinska Institutet identified antisense oligonucleotide therapies as a new possible treatment option for progeria. In mice and human cells, the treatments showed promising results by reducing telomeric non-coding RNA and improving cell division.
A novel method uses subcellular fractionation to quantify unconjugated AONs in nuclei, showing proportional relationship with target gene knockdown. Researchers report their results in Nucleic Acid Therapeutics, highlighting the importance of accurately quantifying oligonucleotides in therapeutic applications.
Researchers proposed new guidelines to standardize experiments with synthetic nucleic acids, addressing current issues like inadequate controls and misleading data interpretation. The guidelines focus on practical advice for performing experiments, measuring gene expression, and selecting appropriate controls.
A team of researchers at the University of Pittsburgh has developed a method to trap the pathological protein TDP-43, which is linked to 97% of amyotrophic lateral sclerosis cases. An oligonucleotide-based approach prevents TDP-43 aggregation and rescues neurons from neurodegeneration.
Researchers found a way to reduce telomerase activity in cancer cells by using specific oligonucleotides, which may lead to new antitumor drugs. The study showed that the combination of oligonucleotides reduced telomerase activity by 10 times within 24 hours.
A first-in-human study demonstrated the ability of a new class of antisense oligonucleotide therapeutics to target the liver, resulting in improved potency and safety at therapeutic doses. The study showed that the conjugated drug was up to 30-fold more potent than the parent antisense oligonucleotide.
Researchers have developed a two-pronged approach targeting Ebola virus infection using linked nucleic acid (LNA) antisense oligonucleotides (ASOs). The study demonstrates effective targeting of two Ebola viral genes and the ability to prevent infectivity via Niemann-Pick C1.
Researchers have identified a range of synthetic antisense oligonucleotides that can activate the frataxin gene in FA patients. The discovery demonstrates flexible options for increasing frataxin protein expression and alleviating FA symptoms.
The approval of Spinraza marks the emergence of a new class of biological products, following predictable patterns of innovation. Analysis of over 60,000 research publications reveals that successful product development characteristically follows the maturation of enabling research.
Researchers have developed a new method to stabilize small interfering RNAs (siRNAs) by introducing phosphoramidate modifications, which enhances their stability and therapeutic potential. The study shows that the modified siRNAs maintain their gene silencing activity, making them suitable for various therapeutic applications.
A study of oligonucleotide drug impurities reveals opportunities for process improvements and the application of information from one drug substance to another. The report, published in Nucleic Acid Therapeutics, provides clarity on product-related impurities.
Scientists develop a targeted therapy that slows disease progression in mice with ataxia and ALS, suggesting potential benefits for humans. The treatment targets the ataxin-2 gene, which may also be important for maintaining brain cell health.
Researchers have discovered a potential treatment for ALS and spinocerebellar ataxia 2 using gene silencing technology. The drug, an antisense oligonucleotide, reduces problems associated with SCA2 by silencing the ataxin 2 gene.
A study at Stanford University revealed that suppressing a specific protein in a mouse model of ALS extends the animal's lifespan. Researchers found that mice treated with a drug designed to block the production of this protein lived over 400 days, while untreated mice died within 29 days.
Researchers have developed a DNA storage method called DNA Fountain that approaches the theoretical maximum for DNA storage, storing 60% more data than previous efforts. The technique uses a coding approach to randomly package information and reassemble it in order, minimizing errors and allowing for reliable retrieval of stored data.
Researchers found a potential treatment for Alzheimer's disease and other neurodegenerative disorders using a designer compound that prevents tau protein damage. The compound, called tau antisense oligonucleotides, was shown to reverse brain injury in mice and monkeys.
Dr. Stanley T. Crooke presents a detailed look at the mechanisms underlying antisense drug activity, including prehybridization, hybridization, and post-hybridization phases. He highlights his group's contributions to characterizing ASO therapeutics' design, targeting, and mechanism of action.
Researchers develop antisense oligonucleotide to lower tau protein levels in mice, reversing neurological damage and improving survival. The treatment also shows promise in monkeys, suggesting a potential therapeutic approach for Alzheimer's and other tau-related diseases.
Researchers have developed single-stranded silencing RNAs (ss-siRNAs) with improved potency and activity, using inexpensive chemical modification to enhance their therapeutic potential. The study's findings have the potential to democratize gene therapy research, enabling more researchers to explore new treatments.
Researchers propose that certain individuals may experience heightened sensitivity to nanoparticles due to pre-existing conditions affecting the liver or lungs. This suggests a need for personalized models and clinical trials to better understand nanoparticle-related infusion reactions.
A team of scientists has discovered a mechanism by which tumor cells elevate levels of MDM4, a protein highly expressed in cancer cells. Targeting MDM4 abundance with antisense oligonucleotides (ASOs) impairs tumour growth and increases cell death, offering a promising clinically-compatible therapeutic target for various cancers.